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New breakthrough in cancer research promises better immune therapy – Health24

In 2017 we reported on an experimental immunotherapy cancer treatment which showed extraordinary results in clinical trials. This type of treatment is known as T-cell cancer therapy.

How exactly does T-cell cancer therapy work?

The field of immunotherapy aims to "supercharge" the body's own immune system, better enabling it to fight cancer.

T-cells are white blood cells that form part of the immune system, detecting and attacking abnormal or foreign cells in the body.

In cancer patients, T-cells normally aren't able to completely rid the body of cancer.

In T-cell therapy, immune cells are engineered to better recognise and fight cancer cells. Researchers extract these cells from blood and edit them with gene transfers to produce a potent receptor that can more effectively fight cancer. The cells are then placed back into the patients body

While this type of treatment was initially used in patients with an advanced form of leukaemia, a new breakthrough meant that T-cell therapy could be used for many other types of cancer.

What does the new research entail?

According to a news report, in 2020, researchers discovered a new immune cell receptor. This means that T-cell therapy as we know it can work much, much better and fight more cancers as it was only able to recognise a handful of cancers in the past.

This meant that, because of a T-cell receptor called human leukocyte antigen (HLA), the treatment had to be personalised for every single patient. This is the receptor that enables the cells to detect and fight the cancer.

Usually HLA varies from person to person, but that is where the new discovery could be a breakthrough.

The new study was led by scientists from Cardiff University in the UK who used the CRISPR-Cas9 screening to discover a new type of receptor called MR1. The full study can be found in the journal Nature Immunology.

These receptors do exactly what HLA does, but there is one big difference the treatment doesnt differ from person to person, resulting in a much better basis for T-cell therapy.

Too soon to tell, but prospect is exciting

There is, however, one caveat. The research is still very new and was conducted in a lab, which means that positive results first need to be achieved in clinical trials.

Experts remain optimistic and call this discovery an exciting new frontier.

"This research represents a new way of targeting cancer cells, which is really quite exciting, although much more research is needed to understand precisely how it works,"says research and policy director Alasdair Rankin from the blood cancer charity Bloodwise, who was not involved in the research

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New breakthrough in cancer research promises better immune therapy - Health24

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Global Healthcare Market Outlook, 2020: Key Predictions & Growth Opportunities for the $2+ Trillion Industry – Yahoo Finance

Dublin, Jan. 23, 2020 (GLOBE NEWSWIRE) -- The "Global Healthcare Market Outlook, 2020" report has been added to's offering.

Amid rising global trade tensions and sluggish global economic outlook for 2020, the global healthcare market is expected to cross the $2 trillion mark in 2020.

Healthcare will be among the top two priorities for voters in the 2020 presidential election in the US. In the European region, looming BREXIT indecision is likely to have a strong impact on Europe's biggest digital health market (UK). Globally, 2020 will be a reality check for long-pending national healthcare policies and regulatory reforms that must re-invigorate future strategies.

The new vision for healthcare for 2020 and beyond will not just focus on access, quality, and affordability but also on predictive, preventive, and outcome-based care models promoting social and financial inclusion. Social Determinants of Health (SDOH) will emerge has a big theme across progressive health systems to proactively engage the right patients and improve health outcomes to help healthcare organizations meet quality standards. In 2020, consumer-driven models of healthcare will gain more market traction, as they stand to better bridge the gap of what consumers want and what healthcare can deliver.

Continued steps will be taken by retail (Walmart, Costco, Amazon, Ali Health), and consumer tech (Google, Apple, Microsoft, and so on) companies globally; to make further headway (intrude) into vetted healthcare space. In 2020, the convergence of Artificial Intelligence (AI), Blockchain, and the Internet of Things (IoT) will further catalyze the space of innovation adoption and related applications in the healthcare realm. For example, while Blockchain will improve data liquidity to empower AI and analytics vendors/applications to digest a large amount of data, AI can manage Blockchain systems more efficiently than humans.

Research Scope

Every year, the team of futurists, analysts, and consultants at the publisher's Transformational Healthcare Group come together to render a comprehensive analysis to predict the themes, technologies, and global forces that will define the next 12 to 18 months (future) for the healthcare industry.

As a part of this research deliverable, the publisher provides bold perspectives and predictions for the global healthcare market in 2020. The sectors covered include pharmaceuticals and biotech, in-vitro diagnostics, medical technologies, medical imaging, and healthcare IT. The analysis captures sectoral and regional trends and provides predictions for the upcoming year. The study provides guidance on where to find the greatest opportunities for expansion.

Predictions for the global healthcare market in 2020 include:

Key Issues Addressed

Key Topics Covered

1. Executive Summary

2. Revisiting 2019 Predictions

3. Global Healthcare Market Outlook for 2020

4. Key 2020 Healthcare Market Predictions

5. Regional Predictions 2020

6. Sector Outlook 2020

7. Key Conclusions

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Global Healthcare Market Outlook, 2020: Key Predictions & Growth Opportunities for the $2+ Trillion Industry - Yahoo Finance

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Genprex rakes in $8M in at-the-market stock offering – Proactive Investors USA & Canada

The gene therapy company sold 7.6 million shares of stock at a price of $1.05 per share

Genprex Inc () announced an $8 million offering of stock through securities purchase agreements made with influential investors.

The gene therapy company sold 7.6 million shares at a price of $1.05 per share, in accordance with Nasdaq rules governing registered direct offerings.

Genprex, based in Austin, Texas and Cambridge, Massachusetts, plans to use the proceeds to advance its clinical programs in non-small cell lung cancer, as well as for working capital and general corporate purposes.

The company is developing a new approach to treating cancer, including its initial product candidateOncoprex, which is an immunogene therapy for non-small cell lung cancer.

Contact Andrew Kessel at [emailprotected]

Follow him on Twitter @andrew_kessel

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R&D Activities to Fast-track the Growth of the Gene Therapy and Antisense Drugs Market Between 2017 2025 – Fusion Science Academy

The Gene Therapy and Antisense Drugs Market research report added by Market Study Report, LLC, provides a succinct analysis on the recent market trends. In addition, the report offers a thorough abstract on the statistics, market estimates and revenue forecasts, which further highlights its position in the industry, in tandem with the growth strategies adopted by leading industry players.

The Gene Therapy and Antisense Drugs market study is a well-researched report encompassing a detailed analysis of this industry with respect to certain parameters such as the product capacity as well as the overall market remuneration. The report enumerates details about production and consumption patterns in the business as well, in addition to the current scenario of the Gene Therapy and Antisense Drugs market and the trends that will prevail in this industry.

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What pointers are covered in the Gene Therapy and Antisense Drugs market research study?

The Gene Therapy and Antisense Drugs market report Elucidated with regards to the regional landscape of the industry:

The geographical reach of the Gene Therapy and Antisense Drugs market has been meticulously segmented into United States, China, Europe, Japan, Southeast Asia & India, according to the report.

The research enumerates the consumption market share of every region in minute detail, in conjunction with the production market share and revenue.

Also, the report is inclusive of the growth rate that each region is projected to register over the estimated period.

The Gene Therapy and Antisense Drugs market report Elucidated with regards to the competitive landscape of the industry:

The competitive expanse of this business has been flawlessly categorized into companies such as


On the basis of therapeutic area, the gene therapy and antisense drugs market is segmented into cancer, anemia, rheumatoid arthritis, cardiovascular diseases, HIV/AIDS, cystic fibrosis, diabetes mellitus and obesity, and renal diseases.

By gene transfer method, ex vivo gene transfer and in vivo gene transfer are the segments of the market. The former involves the transfer of cloned genes into cells, i.e., cells are altered outside the body before being implanted into the patient, whereas the latter involves the transfer of cloned genes directly into the patients tissues. The outcome of in vivo gene transfer technology mainly depends on the general efficacy of gene transfer and expression.

Global Gene Therapy and Antisense Drugs Market: Regional Outlook

The global gene therapy and antisense drugs market is segmented into North America, Asia Pacific, Europe, and Rest of the World. Amongst these, North America holds the leading position in the market followed by Europe. The increasing incidence of cancer and other fatal diseases, unhealthy lifestyle practices such as excessive smoking and excessive consumption of high fat content food, and increasing research efforts for treatment against cancer are the major factors driving the gene therapy and antisense drugs market in these regions.

Asia Pacific is expected to emerge as a significant market for gene therapy and antisense drugs. The high population density including a large geriatric population, expeditiously increasing demand for technologically advanced therapeutics, and increasing government support for improved healthcare infrastructure in the region is driving the growth of this regional market. Furthermore, favorable reimbursement policies and tax benefits on newer therapies will further fuel the growth of the Asia Pacific gene therapy and antisense drugs market.

Major Companies Mentioned in Report

Some of the leading companies operating in the global gene therapy and antisense drugs market are GenVec Inc., Avigen Inc., Genome Therapeutics Corp., Tekmira Pharmaceuticals Corporation, Isis Pharmaceuticals, Cell Genesys Inc., and others. These companies are profiled for their key business attributes in the report.

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Exclusive details pertaining to the contribution that every firm has made to the industry have been outlined in the study. Not to mention, a brief gist of the company description has been provided as well.

Substantial information subject to the production patterns of each firm and the area that is catered to, has been elucidated.

The valuation that each company holds, in tandem with the description as well as substantial specifications of the manufactured products have been enumerated in the study as well.

The Gene Therapy and Antisense Drugs market research study conscientiously mentions a separate section that enumerates details with regards to major parameters like the price fads of key raw material and industrial chain analysis, not to mention, details about the suppliers of the raw material. That said, it is pivotal to mention that the Gene Therapy and Antisense Drugs market report also expounds an analysis of the industry distribution chain, further advancing on aspects such as important distributors and the customer pool.

The Gene Therapy and Antisense Drugs market report enumerates information about the industry in terms of market share, market size, revenue forecasts, and regional outlook. The report further illustrates competitive insights of key players in the business vertical followed by an overview of their diverse portfolios and growth strategies.

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Some of the Major Highlights of TOC covers:

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R&D Activities to Fast-track the Growth of the Gene Therapy and Antisense Drugs Market Between 2017 2025 - Fusion Science Academy

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Global Cake Mix Market Industry: A Latest Research Report to Share Market Insights and Dynamics – Fusion Science Academy

This report presents the worldwide In Situ Hybridization market size (value, production and consumption), splits the breakdown (data status 2018 and forecast to 2025), by manufacturers, region, type and application.

This study also analyzes the market status, market share, growth rate, future trends, market drivers, opportunities and challenges, risks and entry barriers, sales channels, distributors and Porters Five Forces Analysis.

The report presents the market competitive landscape and a corresponding detailed analysis of the major vendor/key players in the market.

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Top Companies in the Global In Situ Hybridization Market:

key drivers, restraints, recent trends, and growth opportunities in the global market for in situ hybridization. The market shares, product portfolios, technological developments, and business strategies of the major players are discussed in detail. The report also sheds light on factors such as market size, supply and demand ratio, market attractiveness, and key segments of the in situ hybridization market.

Global In Situ Hybridization Market: Drivers and Restraints

Technological advancements in the field of in situ hybridization, such as development of the cytogenetic technique, is one of the key factors driving the market. The rising prevalence of cancer has necessitated clinical research, which is another major market driver. The introduction of new therapeutic agents has led to a high rate of adoption of companion diagnostics, boosting the overall market expansion. Besides these factors, the growth of the pharmaceutical and biotechnology industry has improved the prospects of the in situ hybridization market worldwide.

Florescence in situ hybridization (FISH) can be used in the detection of genetic abnormalities such as aneuploidy, characteristic gene fusion, or loss of a chromosomal region. It is also useful for research in the fields of gene mapping and identification of genetic aberrations, which are responsible for cancer. As this technique is simple yet effective, FISH will ensure the growth of the global in situ hybridization market.

On the contrary, strict regulatory policies will obstruct the growth of the in situ hybridization market. However, the emergence of molecular cytogenetics will present significant opportunities due to unmet needs in accurate disease diagnosis, rising number of chromosomal disorders, and surge in population.

Global In Situ Hybridization Market: Regional Outlook

On the basis of geography, the global market for in situ hybridization can be segmented into Europe, Asia Pacific, Latin America, North America, and the Middle East and Africa. North America holds a large share in the overall market, with Europe and Asia Pacific also exhibiting promising growth. Extensive research activities in countries such as Canada and the U.S., increased adoption of companion diagnostics, and beneficial government policies have been aiding the in situ hybridization market in North America.

Over the forecast period 2017-2025, Asia Pacific will undergo tremendous growth on account of growing incidence of cancer and its diagnosis, increased healthcare expenditure, and increasing health awareness among people. The presence of international companies in countries such as India and China will further provide an impetus to the market.

Companies Mentioned in the Report

The major companies operating in the market for in situ hybridization include Bio Sb, Inc., Advanced Cell Diagnostics, Inc., Merck KGaA, Abbott Laboratories, Inc., Leica Biosystems Nussloch GmbH, Danaher Corporation, and Agilent Technologies. Several companies are using business strategies such as product enhancement, collaborations, acquisitions, and partnerships with a view to increasing profit.

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The report provides a valuable source of insightful data for business strategists and competitive analysis of In Situ Hybridization Market. It provides the In Situ Hybridization industry overview with growth analysis and futuristic cost, revenue and many other aspects. The research analysts provide an elaborate description of the value chain and its distributor analysis. This Tire In Situ Hybridization study provides comprehensive data which enhances the understanding, scope and application of this report.

Influence of the In Situ Hybridization market report:

-Comprehensive assessment of all opportunities and risk in the In Situ Hybridization market.

In Situ Hybridization market recent innovations and major events.

-Detailed study of business strategies for growth of the In Situ Hybridization market-leading players.

-Conclusive study about the growth plot of In Situ Hybridization market for forthcoming years.

-In-depth understanding of In Situ Hybridization market-particular drivers, constraints and major micro markets.

-Favorable impression inside vital technological and market latest trends striking the In Situ Hybridization market.

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The report has 150 tables and figures browse the report description and TOC:

Table of Contents

1 Study Coverage

1.1 In Situ Hybridization Product

1.2 Key Market Segments in This Study

1.3 Key Manufacturers Covered

1.4 Market by Type

1.4.1 Global In Situ Hybridization Market Size Growth Rate by Type

1.4.2 Hydraulic Dredges

1.4.3 Hopper Dredges

1.4.4 Mechanical Dredges

1.5 Market by Application

1.5.1 Global In Situ Hybridization Market Size Growth Rate by Application

2 Executive Summary

2.1 Global In Situ Hybridization Market Size

2.1.1 Global In Situ Hybridization Revenue 2014-2025

2.1.2 Global In Situ Hybridization Production 2014-2025

2.2 In Situ Hybridization Growth Rate (CAGR) 2019-2025

2.3 Analysis of Competitive Landscape

2.3.1 Manufacturers Market Concentration Ratio (CR5 and HHI)

2.3.2 Key In Situ Hybridization Manufacturers In Situ Hybridization Manufacturing Base Distribution, Headquarters Manufacturers In Situ Hybridization Product Offered Date of Manufacturers Enter into In Situ Hybridization Market

2.4 Key Trends for In Situ Hybridization Markets & Products

3 Market Size by Manufacturers

3.1 In Situ Hybridization Production by Manufacturers

3.1.1 In Situ Hybridization Production by Manufacturers

3.1.2 In Situ Hybridization Production Market Share by Manufacturers

3.2 In Situ Hybridization Revenue by Manufacturers

3.2.1 In Situ Hybridization Revenue by Manufacturers (2019-2025)

3.2.2 In Situ Hybridization Revenue Share by Manufacturers (2019-2025)

3.3 In Situ Hybridization Price by Manufacturers

3.4 Mergers & Acquisitions, Expansion Plans

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Global Cake Mix Market Industry: A Latest Research Report to Share Market Insights and Dynamics - Fusion Science Academy

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Gene Therapy Market Is Thriving Worldwide with major players Like: UniQure NV Bluebird Bio, Celladon, Avalanche Bio – Expedition 99

The Global Gene Therapy Market is growing at an exciting pace driven by changing dynamics and risk ecosystem, a study of which forms the crux of the report. The study on the global Gene Therapy market takes a closer look at several regional trends and the emerging regulatory landscape to assess its prospects. The critical assessment of the numerous growth factors and breaks in the global Gene Therapy market offered in the analyses helps in assessing the lucrativeness of its key segments.

Download Free PDF Brochure for Latest Research Study of Gene Therapy Market:: #request_sample

This Report Covers Leading Companies Associated in Worldwide Market:

UniQure NVBluebird Bio,Celladon,Avalanche Bio,Sangamo,Shanghai Sunway Biotech Co. LtdAdvantagene,Sibiono GeneTech,Spark Therapeutics,Dimension Therapeutics,

Summary of Market: The global Gene Therapy market is valued at xx million US$ in 2019 is expected to touch xx million US$ by the close of 2025, growing at a CAGR of xx% during 2020-2025.

The report emphases on Gene Therapy Market volume and value at Global Level, Regional Level And Company Level. From a global standpoint, this report embodies overall market size by studying historical data and future outlook.

The report is bifurcated into product type, applications, and regions worldwide. The above areas are further bifurcated into country-level data statistics for the below countries.

The key regions and countries covered in this report are:

Please note, the regional and country level data can be altered and provided as per clients custom requirements.

Global Gene Therapy Market Segmentation, By Product Type:

Viral vectorNon-viral vector

Global Gene Therapy Market Segmentation, By Application:Oncological DisordersRare DiseasesCardiovascular DiseasesNeurological DisordersInfectious diseaseOther Diseases

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Research objectives:

To study and estimate the market size of Gene Therapy , in terms of value.

To find development and challenges for the global market.

To observe worthwhile expansions including expansions, new services presents in worldwide industry.

To classify and assess the side view of important companies of Global Gene Therapy industry.

Key Questions Answered in the Report:

How is the Gene Therapy market expected to Grow In Terms Of Value during the study period?

What are the Competition Developments and Trends in the Gene Therapy market?

What are the core Macro-Economic and Industry Factors impacting the growth of the Gene Therapy market?

What are the Key Challenges, Opportunities, and Improvements faced by market players in the global Gene Therapy market?

Table of Content

1 Report Outline

1.1 Research Opportunity

1.2 Major Industrialists

1.3 Market Segment by Type

1.4 Market Segment by Application

1.5 Study Objectives

1.6 Years Considered

2 Global Evolution Trends

2.1 Production and Volume Analysis

2.1.1 Global Gene Therapy Production Value 2015-825

2.1.2 Global Gene Therapy Production 2015-2025.

2.1.3 Global Gene Therapy Capacity 2015-2025.

2.1.4 Global Gene Therapy Marketing Pricing and Trends

2.2 Major Producers Growth Rate (CAGR) 2020-2025.

2.2.1 Global Gene Therapy Market Size CAGR of Major Regions

2.2.2 Global Gene Therapy Market Share of Major Regions

2.3 Industry Trends

2.3.1 Market Top Trends

2.3.2 Market Operators

3 Market Share by Industrialists

3.1 Capacity and Production by Industrialists

3.1.1 Global,Gene Therapy Capacity by Industrialists

3.1.2 Global Gene Therapy Production by Industrialists

3.2 Revenue by Industrialists

3.2.1. Gene Therapy Revenue by Industrialists (2015-2020)

3.2.2. Gene Therapy Revenue Share by Industrialists (2015-2020)

3.2.3 Global Gene Therapy Market Concentration Ratio (CR5 and HHI)

3.3. Gene Therapy Price by Industrialists

3.4 Major Industrialists of Gene Therapy Plants/Factories Distribution and Area Served

3.5 Date of Major Industrialists Enter into Gene Therapy Market

3.6 Major Industrialists Gene Therapy Product Offered

3.7 Mergers & Acquisitions, Expansion Plans

4 Market Dimensions by Type

4.1 Production and Production Rate for Each Type

4.2 Global Gene Therapy Production Market Share by Type

4.3 Global Gene Therapy Production Value Market Share by Type

4.4. Gene Therapy Ex-factory Price by Type

5 Market Size by Application

5.1 Overview

5.2 Global Gene Therapy Consumption by Application

6 Production by Regions

6.1 Global Gene Therapy Production (History Data) by Regions 2015-2020.

6.2 Global Gene Therapy Production Value (History Data) by Regions

6.3 North America

6.3.1 North America Production Growth Rate 2015-2020.

6.3.2 North America Production Value Growth Rate 2015-2020.

6.3.3 Major Players in North America

6.3.4 North America Import & Export

6.4 Europe

6.4.1 Europe Production Growth Rate 2015-2020.

6.4.2 Europe Production Value Growth Rate 2015-2020.

6.4.3 Major Players in Europe

6.4.4 Europe Import & Export

6.5 China

6.5.1 China Production Growth Rate 2015-2020.

6.5.2 China Production Value Growth Rate 2015-2020.

6.5.3 Major Players in China

6.5.4 China Import & Export

6.6 Japan

6.6.1 Japan Production Growth Rate 2015-2020.

6.6.2 Japan Production Value Growth Rate 2015-2020.

6.6.3 Major Players in Japan

6.6.4 Japan Import & Export

7. Gene Therapy Consumption by Regions

7.1 Global Gene Therapy Consumption (History Data) by Regions

7.2 North America

7.2.1 North America Consumption by Type

7.2.2 North America Consumption by Application

7.2.3 North America Consumption by Countries

7.2.4 United States

7.2.5 Canada

7.2.6 Mexico

7.3 Europe

7.3.1 Europe Consumption by Type

7.3.2 Europe Consumption by Application

7.3.3 Europe Consumption by Countries

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Gene Therapy Market Is Thriving Worldwide with major players Like: UniQure NV Bluebird Bio, Celladon, Avalanche Bio - Expedition 99

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Gene Therapy Market Segmentation 2020 | Analyzing the Impact Followed by Restraints till 2029 – News Monitoring

A recent market study published by in its upcoming report outlook titled, Gene Therapy Market: Global Industry Analysis and Astonishing Growth [2020-2029] offers key market insights and emerging trends on the global Gene Therapy market.

The report has offered an exhaustive analysis of the Gene Therapy Market taking into consideration all the crucial aspects like growth factors, constraints, market developments, and future prospects. Market researchers and industry experts have pointed out the key market trends and prospects that may impact the overall Gene Therapy Market growth. This will help players to leverage the opportunities to strengthen their position. Also, the report throws light on the important factors that are contributing to the Gene Therapy Market growth. Additionally, challenges and impeding factors that could hamper the growth of the Gene Therapy Market in the years to come are mentioned in the report.

The Gene Therapy market report includes comprehensive information about the markets major competitors, including various organizations, companies, associations, suppliers and manufacturers competing for production, supply, sales, revenue generation, and after-sales performance expectations. The bargaining power of numerous vendors and buyers have also been included in the research report.

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Following Key Players are Analysed in this Report: Novartis, Kite Pharma Inc, GlaxoSmithKline PLC, Spark Therapeutics Inc, Bluebird bio Inc, Genethon, Transgene SA, Applied Genetic Technologies Corporation, Oxford BioMedica PLC, NewLink Genetics Corp., Amgen Inc

Market Dynamics:

Set of qualitative informative data that incorporates PORTER Five Forces Model, Macro-Economic factors, PESTEL Analysis, SWOT Analysis, Value Chain Analysis, Regulatory Framework along with Industry Background and Overview.

Worldwide Gene Therapy Research Methodology presents a detailed picture of the market by way of study, synthesis, and summation of information from various sources. The information thus presented is reliable, comprehensive, and the result of extensive research, both primary and secondary. The analysts have presented the different features of the market with a particular focus on identifying the key business influencers.

Gene Therapy Market Data Break Down is illuminated below by vector type, gene type, application, and region:

By Vector:Viral vectorRetrovirusesLentivirusesAdenovirusesAdeno Associated VirusHerpes Simplex VirusPoxvirusVaccinia VirusNon-viral vectorNaked/Plasmid VectorsGene GunElectroporationLipofection

By Gene Therapy:AntigenCytokineTumor SuppressorSuicideDeficiencyGrowth factorsReceptorsOther

By Application:Oncological DisordersRare DiseasesCardiovascular DiseasesNeurological DisordersInfectious diseaseOther Diseases

The report cast light on an extensive analysis based on the most prominent Gene Therapy manufacturers operating in the industry. Activities performed by robust Gene Therapy manufacturers/companies are product development, research, and innovation as well as technology adoptions that are intensifying the competitive intensity and companys ability to offer better product lineup. The companies are also performing strategic acquisitions, ventures, mergers, and partnerships to enlarge their serving area and strengthen their existence worldwide.

The study delivers an exact evaluation of the financial operations of companies covering Gene Therapy sales volume, capital investment, gross margin, profitability, revenue, cash flow, and growth rate. Their manufacturing capacity, production volume, product specifications, import-export activities, production processes, raw material sourcing, and distribution networks are also elaborated in this Gene Therapy report. Companies are also engaged in product launches, promotional activities, and brand developments as part of strategic planning.

How will the report help new companies to plan their investments in the Gene Therapy market?

The Gene Therapy market research report classifies the competitive spectrum of this industry in elaborate detail. The study claims that the competitive reach spans the companies.

The report also mentions about the details such as the overall remuneration, pricing trends, product sales figures, gross margins, etc.

Information about the sales & distribution area alongside the details of the company, such as company overview, product specifications, buyer portfolio, etc., are provided in the Gene Therapy market study.

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Regional Outlook: Regional analysis is another important part of the report which is segregated into different sections. One section of the report is entirely dedicated to regional consumption analysis whereas another for regional production analysis. It includes North America, Europe, China, Japan, Southeast Asia, India.

Some of the Major Highlights of TOC covers in Gene Therapy Market Report:

Chapter 1: Methodology & Scope of Gene Therapy Market

Definition and forecast parameters

Methodology and forecast parameters

Data Sources

Chapter 2: Executive Summary of Gene Therapy Market

Business trends

Regional trends

Product trends

End-use trends

Chapter 3: Gene Therapy Industry Insights

Industry segmentation

Industry landscape

Vendor matrix

Technological and innovation landscape

Chapter 4: Gene Therapy Market, By Region

Chapter 5: Company Profile

Business Overview

Financial Data

Product Landscape

Strategic Outlook

SWOT Analysis


Analysts with high skill in information gathering and governance use industry strategies to collate and examine data at all stages. Our analysts are trained to combine superior research methodology, modern data collection techniques, subject expertise and years of collective experience to deliver useful and accurate research reports.

We will be happy to attend to your queries regarding the above-market study. Kindly contact us atinquiry@marketresearch.bizto learn more about the market report.

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Gene Therapy Market Segmentation 2020 | Analyzing the Impact Followed by Restraints till 2029 - News Monitoring

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Cell and Gene Therapy Market to Exceed Revenues Worth US$ By the End of 2020 2027 – Melanian News

Coherent Market Insights has recently announced the addition of a new research report to its repository named, Global Cell and Gene Therapy Market Status and Forecast 20192026, covering top-line subjective and quantitative synopsis data. The market review provides an eccentric tool for analyzing the market in terms of strengths, and weakness, marking opportunities, and supporting strategic and proficient decision-making. The key drivers and restraints affecting the growth of the Cell and Gene Therapy are stated.

A comprehensive analysis of the Cell and Gene Therapy market is presented in this document, along with a brief overview of the segments in the industry. The study presents a feasible estimate of the current market scenario, including the Cell and Gene Therapy market size with regards to the volume and renumeration. The report is a collection of significant data related to the competitive landscape of the industry. It also contains data with regards to several regions that have successfully established its position in the Cell and Gene Therapy market.

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The Top players including:Amgen, Biogen, BioMarin Pharmaceuticals, Bristol-Myers Squibb Company, GlaxoSmithKline, Novartis, Pfizer, Regeneron Pharmaceuticals and Sanofi, Spark Therapeutics, Agilis Biotherapeutics, Angionetics AVROBIO, Freeline Therapeutics, Horama, MeiraGTx, Myonexus Therapeutics, Nightstar Therapeutics, Kolon TissueGene, Inc., JCR Pharmaceuticals Co., Ltd., and MEDIPOST.

The report also points out the latest trends in the market and the various opportunities for the market to grow in the near future. Insightful information about the key players such as business market overview, product offerings, and industry revenue segmentation has been provided in the report. It also serves an extensive analysis of different sections and sub-segments which offers market insights toward the historic market scenarios along with future growth and prospect.

Our competitor profiling includes evaluation of distribution channels and products and services offered by and financial performance of companies operating in the global Cell and Gene Therapy market. We also provide Porters Five Forces, PESTLE, and SWOT analysis to assess competitive threat and examine other aspects of the global Cell and Gene Therapy market. The report offers strategic recommendations, competitor benchmarking for performance measurement, and analysis of partnership, merger, and acquisition targets and industry best practices. It also provides analysis of profitability and cost across the industry value chain.

Competitive Rivalry: The Cell and Gene Therapy report incorporates the detailed analysis of the leading organizations and their thought process and what are the methodologies they are adopting to maintain their brand image in this market. The report aides the new bees to understand the level of competition that they need to fight for to strengthen their roots in this competitive market.

Principal Research:

The research team works with industry experts from the Global Cell and Gene Therapy industry including the management organizations, processing organizations, value chain analytics by service providers of the Cell and Gene Therapy market.

Subordinate Research:

In the Secondary research vital information about the Cell and Gene Therapy industries value chain, total pool of key players, and application areas. Market separation is done as per the industrial drifts to the deepest level, terrestrial markets and key developments from both market place and technology-oriented viewpoints.

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This report forecasts revenue growth at a global, regional & country level, and provides an analysis of the market trends in each of the sub-segments from 2019 to 2026.

North America (USA, Canada, Mexico, etc.)

Asia-Pacific (China, Japan, India, Korea, Australia, Indonesia, Taiwan, Thailand, etc.)

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Cell and Gene Therapy Market to Exceed Revenues Worth US$ By the End of 2020 2027 - Melanian News

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French-Belgian biotech banks 20M to break ground in blood-splattered field of therapeutic cancer vaccines – Endpoints News

About a decade ago, the ill-fated therapeutic cancer vaccine Provenge was approved, eventually bankrupting its developer Dendreon. Since then, a number of drugmakers have seen similar efforts splutter and fizzle, although the emergence of immunotherapies checkpoint inhibitors and CAR-T drugs offered a glimmer of hope in resuscitating the field. Banking on that promise is PDC*line Pharma, which secured a 20 million injection on Wednesday.

Spun out of the French Blood Bank, the Belgian-French biotech is developing off-the-shelf cancer vaccines that are based on a therapeutic cell line of plasmacytoid dendritic cells, which are sometimes referred to as the Swiss army knife of immune cells due to their diverse range of function. PDC*line Pharmas technology is being developed to be synergistic with checkpoint inhibitors.

Scientists initially hoped that turbocharging the immune system to battle cancer cells would be enough to shrink tumors. But that didnt quite occur since cancer cells possess the ability to put brakes on that immune assault.

In the case of Provenge, data showed the product helped prostate cancer patients live longer but there was no evidence of tumor shrinkage or cancer cell death. Still,Wall Street tagged it with blockbuster expectations. But its adoption was restricted by its complex autologous therapeutic administration, high price tag and changing treatment landscape. Since then, a number of other therapeutic cancer vaccine makers have crashed and burned including Argos Therapeutics and Bavarian Nordic.

A few years ago, the emergence of checkpoint inhibitors which are engineered to unleash the immune system emerged as the perfect partner in crime for the therapeutic cancer vaccine. That is what PDC*line Pharma hopes its vaccine will accomplish.

Its lead product is currently in a Phase I/II study in patients with the most common form of lung cancer. Enrollment is expected to be completed by 2022, chief Eric Halioua told Endpoints News.

Provenges complicated autologous administration, cost of goods, batch-to-batch variability and underwhelming efficacy all contributed to its tepid sales, Halioua suggested, indicating that PDC*line Pharmas plan to incorporate a checkpoint inhibitor into the equation would likely be beneficial.

You need a good vaccine to activate (the) immune system, and you need something to break the defense mechanisms of the tumor. And all the first generations of vaccines were not in these situations.

Although the company is initially testing its off-the-shelf vaccine as a monotherapy, eventually a checkpoint inhibitor will be added to the mix.

But caution is warranted: Efforts to combine therapeutic cancer vaccines and checkpoint inhibitors have also met with failure. Last month, French biotech Transgene abandoned its therapeutic lung cancer vaccine after mid-stage data showed that when tested alongside Bristol-Myers Opdivo, the combination did not significantly shrink tumors.

Altogether PDC*line Pharma which recently signed a 108 million licensing deal (plus royalties) for its lung cancer vaccine in South Korea and other Asian regions has raised more than 30 million since its inception in 2014.

In this latest round of Series B funding, PDC*line Pharma lured five new investors: Korean Investment Partners, as well as two South Korean funds Shinhan-Cognitive Start-up Fund and UTC 2019 BIOVENTUREFUND in addition to two Belgian funds, SRIW (The Regional Investment Company of Wallonia) and Sambrinvest (the investment fund of Charleroi).

The new investors joined existing investors SFPI-FPIM, the Belgian Federal Holding and Investment Company, Noshaq Group, the Financire Spin-off Luxembourgeoise/INVESTSUD Group, among other undisclosed names.

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French-Belgian biotech banks 20M to break ground in blood-splattered field of therapeutic cancer vaccines - Endpoints News

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Discovery of gene that modifies severity of inherited kidney disease – Devdiscourse

Experts at Newcastle University have shown that the rate of kidney disease in people with Joubert syndrome is determined by the genetic makeup of the individual and each patient may respond differently to the treatment.

Joubert syndrome is a complex disorder, affecting approximately one in 80,000 newborns, causing varying degrees of physical, mental and sometimes visual impairments. It is often associated with severe kidney disease that requires dialysis and ultimately transplantation.

The study, published online in the Proceedings of the National Academy of Sciences, is the first time that an explanation has been given for the difference of disease progression in Joubert syndrome patients.

Significant breakthrough

The Newcastle research has identified a second gene called BSND a 'modifier gene' which determines the severity of kidney disease in patients with CEP290 mutations of Joubert syndrome.

It has been assumed that these modifier genes exist, but they have never been found before in rare genetic conditions until now.

Professor John Sayer and Dr Colin Miles, from the Translational and Clinical Research Institute, Newcastle University, led the Medical Research Council-funded research.

Professor Sayer said: "We have shown, using mouse and human DNA samples, that BSND is a modifier gene for the severity of kidney disease in Joubert syndrome.

"This is the first time that a modifier gene for inherited kidney disease has been identified, and this information will improve diagnoses and will be used to develop therapies to reduce the severity of kidney disease in affected patients.

"Our research is a major step forward and, in the future, we may be able to offer a therapy that switches on the protective modifier gene and reduces the development of genetic kidney disease.

"This work paves the way towards personalised therapies in patients with inherited kidney disease."

The international study used mouse models and DNA samples from patients with Joubert syndrome to progress the research.

Scientists used mouse models of disease and genetic manipulation to see how the kidney disease responded to modifier gene manipulation, cross-referenced with DNA sequencing data from patients around the world to prove the modifier gene was relevant in humans.

Challenging disease

Professor Sayer, a Consultant Nephrologist at Newcastle Hospitals NHS Foundation Trust, said: "The treatment of genetic kidney disease is challenging, as this requires both the correction of the underlying gene defect and the delivery of the treatment.

"We have shown that the kidney disease in a mouse can be dramatically changed by switching on or off a modifier gene.

"This will mean that we can use this information to carry out treatments, including genetic therapies, to lessen the effects of inherited kidney diseases, such as Joubert syndrome.

"We are testing these treatments further in our model systems before we move into patient studies."

Within the next three years, research will start to test the treatment of patients with modifier genes in the hope of developing personalized treatment plans.

Patient story

Siblings Emma, 11, and Ben Buckley, eight, have Joubert syndrome and both developed kidney failure before the age of eight.

They were diagnosed with Joubert syndrome from a few months of age and both have required dialysis and a kidney transplant.

They suffer from a range of medical issues due to Joubert syndrome, including visual impairment, communication problems, and developmental delay.

The two children, of Whitley Bay, North Tyneside, have been instrumental in helping further the research over the years, allowing the Newcastle scientists to study the mutation in detail.

Parents Leanne and Michael say they welcome the findings of the Newcastle University-led study as it will help to give patients a chance of preventing kidney failure in the future.

Leanne said: "It is very important that research is done into Joubert syndrome and the linked kidney damage, as this will hopefully prevent patients in the future needing a kidney transplant.

"All throughout Ben and Emma's lives, they have lived with the effects of Joubert syndrome and scientists found they had a problem with the CEP290 gene.

"Both Ben and Emma have needed dialysis and kidney transplants because of their kidney problems and I would like to hope this research will help prevent kidney failure for other affected children.

"We were happy for Ben and Emma to provide samples for the study as anything that helps further understanding into the condition is well worth doing, so it's great to see the study's positive results."

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Discovery of gene that modifies severity of inherited kidney disease - Devdiscourse

Recommendation and review posted by Bethany Smith

Nanoparticle Therapy Shows Promise With Breast Cancer – UMB News

Researchers at theUniversity of Maryland School of Medicine (UMSOM) developed a new nanoparticle drug formulation that targets a specific receptor on cancer cells and appears to be more effective than a standard nanoparticle therapy currently on the market to treat metastatic breast cancer, according to a study published today in the journal Science Advances. The new DART (decreased, non-specific adhesivity, receptor-targeted) nanoparticles bypass healthy cells and tissues and bind to tumor cells, dispersing evenly throughout the tumor while releasing the chemotherapy drug paclitaxel.

The marketed drug Abraxane, a nanoformulation containing paclitaxel that is currently used to treat women with aggressive breast cancer, is an effective agent, but it was not designed to selectively deliver paclitaxel to only the cancerous cells within the body, said study corresponding co-author Jeffrey Winkles, PhD, a professor of surgery at UMSOM. Our DART nanoparticle specifically targets the Fn14 receptor found abundantly on breast cancer cells; it uses this receptor to gain entry through the plasma membrane and deliver the drug to destroy the cancer. Winkles group discovered the Fn14 receptor and described its potential as a target for new therapeutics more than a decade ago.

For this study, UMSOM researchers engineered and tested a new therapeutic nanoparticle platform to deliver the drug paclitaxel to treat triple-negative breast cancer. About one in five women with breast cancer have this type of aggressive tumor, which is particularly difficult to treat; these cancers lack receptors commonly expressed by most breast cancer cells, like hormone receptors, for which effective drugs have been designed. But many triple-negative breast cancers express high levels of Fn14; indeed, most solid tumor types, including lung, prostate, and colorectal cancer, overexpress this cell surface receptor.

After much initial hype and some disappointments in the field, nanoparticle delivery systems for cancer treatment are starting to show real promise for patients, said study co-author Graeme Woodworth, MD, professor and interim chair in the Department of Neurosurgery at UMSOM. We established a proof-of-concept with this study, outlining the design of an optimized nanoparticle delivery system that balances the specific binding to cancer cells while minimizing non-specific, off-target binding to other cells in a very fine-tuned way.

To accomplish this, the research team attached a monoclonal antibody called ITEM 4 to the surface of the nanoparticle because it specifically binds to Fn14, providing a key to unlock entry into the cancer cell. The surface of the nanoparticles also was coated with polyethylene glycol to keep them circulating through the bloodstream and lymph system until they reached the tumor and to prevent them from being quickly flushed out of the body.

Many drug delivery carriers exhibit non-specific binding to healthy cells and tissues in addition to the diseased cells they are targeting, which often leads to unintended side effects or toxicities, said study corresponding co-author Anthony Kim, PhD, associate professor of neurosurgery and pharmacology at UMSOM. This DART nanoparticle platform has unique capabilities to improve therapeutic delivery to difficult-to-treat locations within the body while also allowing us to potentially increase the maximum tolerated dose of the encapsulated drug without increasing side effects to patients.

The researchers filled their optimized DART nanoparticle formulation with paclitaxel and tested it against Abraxane (the marketed nanoparticle that also contains paclitaxel) in animals with triple-negative breast cancer tumors. In one set of experiments, the nanoparticles were delivered to mice harboring breast tumors grown above the natural breast region. They found the DART formulation led to a significantly increased median overall survival (68 days) compared to Abraxane treatment (45 days). They also saw a clear benefit to using the DART nanoparticles when they compared the treatments again in animals that harbored breast tumors implanted in the brain (akin to a metastatic brain tumor).

This is a compelling finding and significant step forward in the use of nanoparticles to treat cancer, said UMSOM Dean E. Albert Reece, MD, PhD, MBA, the John Z. and Akiko K. Bowers Distinguished Professor and University of Maryland, Baltimore executive vice president for medical affairs. It fits squarely with our School of Medicines mission to advance the field of potentially lifesaving therapies for patients with the most difficult to treat cancers.

The UMSOM researchers are members of the University of Maryland Marlene and Stewart Greenebaum Comprehensive Cancer Center. A former PhD student, Jimena Dancy, PhD, and a former postdoctoral fellow, Aniket Wadajkar, PhD, are listed as co-first authors on the publication. Researchers from the Translational Genomics Research Institute in Phoenix, Ariz., and the Mayo Clinic Arizona in Scottsdale, Ariz., also contributed to the work.

This study was primarily funded by the National Institutes of Health.

Future research includes testing the DART therapy in other cancer types, including an aggressive form of brain cancer called glioblastoma and developing a similar version of the nanoparticle designed to work specifically in humans. This would involve using a humanized antibody on the surface of the nanoparticle and scaling up the formulation. The researchers recently received a grant from the TEDCO Maryland Innovation Initiative Commercialization Program to move forward with efforts to adapt their nanoparticle system and eventually test the treatment in cancer patients.

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Nanoparticle Therapy Shows Promise With Breast Cancer - UMB News

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Endometriosis and cannabis: THC improves symptoms – Health Europa

Endometriosis is a painful condition where the lining of the uterus grows on other parts of the organ such as the fallopian tubes. The new study, published in eLife, shows initial results from treating endometriosis in mice with cannabinoids suggesting they can alleviate symptoms of the disease.

The researchers say this new finding will pave the way for further clinical research.

The findings have led to the start of a clinical trial in collaboration with the Gynecology Service of the Hospital Clinic of Barcelona, Spain. The trial will evaluate the possible benefits of the naturally occurring cannabinoid 9-tetrahydrocannabinol (THC; the main psychoactive constituent of the cannabis plant) in women with endometriosis.

Endometriosis is a common, chronic and painful disease caused when the lining of the womb the endometrium grows outside of the womb cavity. These growths affect reproductive organs and can cause pain, infertility, anxiety, depression and result in a considerable impact on quality of life. Treatment options include surgery or hormone therapy, but these are not always effective and often have significant side-effects.

Rafael Maldonado, Professor at the University Pompeu Fabra of Barcelona, Spain, said: With a lack of effective treatments, women with endometriosis usually rely on self-management strategies like dietary changes or exercise. Although cannabis comes with a large number of potential side effects, its medicinal properties could provide pain relief in endometriosis and other conditions.

Since medical THC is available in some countries, the findings of our study may be of interest for gynecologists and pain specialists who manage the treatment of women with endometrial pain.

The team studied mice with endometrial implants in their pelvis to mimic endometriosis in humans. Those with the implants were more sensitive to pain in their pelvis that can also be associated with emotional and cognitive alterations similar to symptoms seen in some women with endometriosis.

The team next found that mice with endometriosis had similar anxiety-like symptoms experienced by some women with the condition. This was measured by the amount of time the animals spent in open areas of a maze, as those with higher anxiety levels tend not to explore too far. However, their experiments could not reveal whether THC had any significant effects in treating this anxiety.

As endometriosis can be known to impair cognitive function in some women, the team also studied memory performance in the mice. They provided the animals with two identical objects and allowed them to become familiar with them. They then replaced one of the objects and timed how long the mice spent exploring the new versus familiar object, to give an indication of what the animals remembered.

The team found that memory was impaired in the mice with endometriosis compared with those that did not have the condition. However, mice treated with THC did not show this impairment, suggesting that THC may have a protective effect.

Finally, the team studied the effects of THC on the endometrium inside and outside of the womb, and found that mice with endometriosis treated with THC for 32 days had smaller endometrial growths.

First author Alejandra Escudero-Lara, a PhD student at the University Pompeu Fabra of Barcelona, said: Together, our findings show that THC limits the development and symptoms of endometriosis in an experimental model, and highlight the interest of conducting further research to ensure the safety and beneficial effects of this treatment in women with endometriosis.

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Endometriosis and cannabis: THC improves symptoms - Health Europa

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Get Rid of Stubborn Belly Fat With 9 Expert Backed Tips – Men’s Health

Stubborn belly fat is more than just an annoyanceit can be dangerous. That's because stomach fat is visceral and surrounds vital organs such as the pancreas and intestines, says Rachel Stahl, R.D. at Weill Cornell Medicine.

It's also biologically active, meaning this type of fat changes the normal balance of hormones and can cause metabolic problems.

"They are not just standard cells storing fat," Stahl tells Men's Health.

Excess belly fat could also be a sign that you have too much fat surrounding your heart, liver, and kidneys, says endocrinologist Dr. Rasa Kazlauskaite, M.D. at Rush University Medical Center in Chicago, IL. When this happens "You are that much closer to developing diabetes," Kazlauskaite tells Men's Health.

So how much belly fat is too much?

Guys who have a waist circumference greater than 40 inches are at a higher risk of diabetes and heart disease, according to the National Institute of Health.

Measure around the middle of your waist, just above the hipbones, using a tape measure, advises the NIH.

Although losing belly fat isn't easy, there are ways to reduce your waistline:

Unfortunately, you can't force fat reduction in one area of your body, says Dr. W. Scott Butsch, M.D., and Director of Obesity Medicine at the Cleveland Clinic.

Instead, you'll want to focus on reducing overall calories, Butsch tells Men's Health.

"Weight loss in general should lead to a decrease in belly fat," he says.

Men's Health

Brain scans show that protein lowers activity in regions of the brain that stimulate food cravings. Protein also increases hormones that make you feel satiated, so you feel fuller for longer periods of time.

They're nutrient dense and provide plenty of fiber, meaning you'll get full on fewer calories. Load up half of your plate with vegetables, or begin every meal with a salad.

Kazlauskaite says guys who want to reduce belly fat should watch how much alcohol they consume. Aside from the obviousempty caloriesalcohol needs to be metabolized by the body, which briefly stops fat burning, she says.

Losing weight shouldn't be your only motivation to hit the gym. And exercise doesn't burn as many calories as you may believe. However, paired with a healthy overall diet, a regular workout routine can help you maintain a calorie deficit.

Clinically, Butsch says many of his patients notice a decrease in belly fat when they incorporate strength training. In fact, obese adolescents who incorporated both aerobic and strength training into their workouts lost the highest amounts of visceral fat, according to a 2014 study published in the Journal of Sports Sciences.

Sugar from juice and sodas are rapidly absorbed into the bloodstream, which spikes insulin, says Kazlauskaite. There's no evidence that these beverages actually cause obesity. However, studies show that frequently drinking sugar-sweetened beverages is linked to weight gain, obesity, and type 2 diabetes, according to the Centers for Disease Control & Prevention.

Eating an apple is different from a drinking a glass of apple juice, explains Kazlauskaite.

That's because you consume more nutrients like fiber when you consume the food in its natural form, she says. Plus, the process of chewing and consuming the food more slowly can help keep you full. "When you eat an apple, you eat slower," she says. "Your body has more time to digest it."

Cortisol, known as the stress hormone, is linked to a higher percent of belly fat and weight gain. In fact, people who have high levels of cortisol for long periods of time are more likely to develop abdominal obesity, according to a 2018 review of studies published in Current Obesity Reports.

Butsch says simple activities like yoga, meditation, or simply avoiding conflict can help keep your waistline trim.

"I think even just taking the higher road in an argument [helps]," he tells Men's Health.

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Get Rid of Stubborn Belly Fat With 9 Expert Backed Tips - Men's Health

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Buses and Uncertainty –

BY Mary E. Burman

The table is loaded with food and the conversation is freewheeling with lots of laughter. Suddenly I remember that I have metastatic breast cancer. I'm feeling generally good with few side effects from my current treatment that hopefully will keep the rogue cells in my bones. At times like this, I can forget that I have metastatic breast cancer which will most likely cause my death. When that will happen is unknowable, and uncertainty is now a constant part of my life. Of course, all of us face uncertainty.

As the saying goes, anybody can walk out the door, get hit by a bus and be dead in seconds. However, as someone with a metastatic disease said, "The only thing I can think of is that I'm going to die. And that's the one certainty that I have, and I know people say () you could cross a street and be run over by a bus. But you live your life not thinking about it. I have to deal with it. My bus is here. All the time. And my bus is outside the door every morning and uncertainty is always with me.

For me, uncertainty manifests itself in several ways. There is a lot of ambiguity about my breast cancer which is "atypical" starting out as hormone positive but is now probably triple negative. It is not as aggressive as I was initially diagnosed with breast cancer 10 years ago. That makes the future unpredictable since most studies don't capture the outcomes for someone like me who is a healthy 60-year-old with an atypical cancer. While metastatic breast cancer is unfamiliar to me, my family has experienced a rare form of ataxia that has resulted in the death of several siblings for most of my adult life I've wondered whether I'd get it. Returning to the bus metaphor, while I was thinking Bus #9 (the ataxia bus) would hit me, it turned out to be Bus #3 (the breast cancer bus) resulting in a new and different uncertainty.

The feeling of uncertainty waxes and wanes and is stressful, fatiguing, intrusive, unending and isolating. Ultimately, uncertainty in metastatic breast cancer is not something you get past. It's always there waiting for the results of tests, figuring out the best treatment, wondering about prognosis and feeling anxious about costs of care. However, paradoxically, uncertainty leaves the door open for hope. Persons with metastatic breast cancer live in two worlds that of uncertainty and that of hope. My future is not written in stone because there is no way of knowing if I will live 2 years or 15 years. Consequently, that leaves space for envisioning the future, e.g., hope for time with my family, hope for being able to accomplish some things, and hope for new treatments.

Simultaneously living with uncertainty while embracing hope is not easy but it can be done. Arthur Brooks explores career transitions in the Atlantic magazine in an article entitled "Your professional decline is coming (much) sooner than you think: Here's how to make the most of it". While not about living with metastatic breast cancer, it a useful framework for living with uncertainty.

He promotes four different strategies for living well. First, jump which means focusing on what is most important in our personal and professional lives and walking away what isn't important. For me that meant stepping out of my administrative roles at work and downsizing to a much smaller and more manageable house and yard. Second, we should find ways to serve and help others. For me, I have renewed my volunteer work for a nonprofit clinic in my community, which provides me much satisfaction. Third, worship by exploring our own spiritual self and I've found great joy in reading, thinking and exploring new realms of spirituality. Finally, Brooks advocates that we connect dedicating time to meaningful and purposeful relationships.

Connecting and reconnecting with others who are comfortable with metastatic breast cancer has been extremely beneficial to me. Ultimately, the bus doesn't leave my street, but it doesn't have to impact my daily life which can be filled with hope, meaningful activities, and relationships despite the uncertainty.

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Breakthrough diabetes insulin drug developed with help from Buffalo – Buffalo News

Some people watch what they eat. Paige and Tanner Szczesekhave to watch when they eat.

The two children, ages 6 and 2, from Cheektowaga, have Type 1 diabetes. Like others with their condition, they must take extra insulin before meal and snacks. It takes time to work. Eat too soon, and blood sugar can climb dangerously high a constant worry for their mother, Ashley.

My biggest goal is that my children can just feel like children, she said.

Thanks to a new drug developed with an assist in Western New York, they have more of a chance.

The Food and Drug Administration last month approved of a drug that brings researchers a step closer to developing an artificial pancreas that will provide fast-acting insulin in proper amounts at just the right time for those with diabetes.

Fiasp, made by Novo Nordisk, starts working in 2 minutes, hits full force within 10 minutes and even can be taken shortly after someone withdiabetesstarts eating.

Previous fast-acting insulins took at least two or three times as long to do that job.

The newest drug has been available to adults since 2017, but the Food and Drug Administration wanted testing on children before making it available to them. More than 700 children in 17 countries participated in the clinical trial, including five children ages 13 to 17 who have been patients in the diabetes centers at UBMD Pediatrics and next door at Oishei Childrens Hospital on the Buffalo Niagara Medical Campus.

This was a big commitment for the families, said Dr. Kathleen E. Bethin, a clinical professor in the Department of Pediatrics in the Jacobs School of Medicine and Biomedical Sciences at the University at Buffalo, and a physician at both diabetes centers. The kids were in the study for almost a year. There were a lot of extra blood draws and more blood sugar checks than are typically required.

Fiasp brings diabetes researchers closer to mimicking a closed-loop system that uses a glucose monitor, continuous insulin infusion pump and other technologies to allow people whose bodies dont make insulin to live like those whose do.

The new fast-acting insulin drug Fiasp boosts the prospects for a closed-loop insulin system often referred to as "an artificial pancreas," diabetes researchers in Buffalo and elsewhere say. (John Hickey/Buffalo News)

The goal in the diabetes field is to develop insulin analogs that behave more like natural insulin, which is rapid on, rapid off, meaning its quickly released, then quickly dissipates, said Dr. Lucy D. Mastrandrea, chief of the Division of Endocrinology/Diabetes at UBMD Pediatrics and medical director for the Oishei Childrens Hospital Diabetes Center. Part of the reason this drug was developed was to have a better timeline of action thats closer to natural insulin.

The body breaks down carbohydrates into blood sugar to use for energy. Insulin is a hormone needed to bring glucose from the bloodstream into human cells. When blood sugar gets too low, the process breaks down and can lead to learning challenges, seizures, loss of consciousness and death. When it spikes, especially often and over time, complications include limb amputation, heart and kidney disease, and stroke.

For those without diabetes, the pancreas, liver and other organs work together to automatically and seamlessly produce insulin and adjust levels as needed.

Type 2 diabetes is diagnosed when the body doesnt use insulin properly. It often can be managed through a combination of healthy eating, regular exercise and oral medications. Sometimes, insulin also is needed.

Those with Type 1 diabetes produce no insulin. They need to inject manufactured basal insulin to maintain levels throughout each day, load carbs or take medication when blood sugar levels get too low, and add fast-acting insulin to lower them when blood sugar levels climb.

Nearly 18,000 new cases of Type 1 diabetes are diagnosed each year. The majority of children have Type 1, while the majority of those diagnosed in adulthood have Type 2.

Our goal is to keep them in as best control as possible during their childhood years, so that they're not running the risk of dealing with complications when they're in their 20s and 30s, Mastrandrea said.

Drug-maker Eli Lilly engineered the first human-derived insulin, Humulin, in 1981. The company in 1996 developed a faster-acting insulin, Humalog, which is still routinely used. NovoLog and Apidra are among other brands that can lower blood sugar within 20 to 30 minutes after they are injected through a needle or an insulin pump, said Dr. Paresh Dandona, a leading international diabetes researcher, head of the Western New York Center of Diabetes-Endocrinology in Amherst and distinguished professor and chief of endocrinology, diabetes and metabolism in the UB medical school.

Administering the drugs takes planning and guesswork because eating, exercise, stress, illness and other factors affect blood sugar levels. That means those with diabetes need to predict related dips and spikes well in advance. Mealtimes generally are the most challenging because they can spark pronounced spikes.

The food hits you a lot faster than the insulin does, said Szczesek.

Fiasp changes the equation. The newer formulation of NovoLog includes niacinamide (vitamin B3) to boost the speed of absorption.

There still is a lag, but it's the best thing we have, said Dandona, whose clinic helped with adult trials several years ago and who has prescribed the drug to some of his patients during the last two years, with good results.

Paige Szczesek, 6, of Cheektowaga, looks at her personal diabetes manager, which helps her and others more closely track her blood glucose levels and insulin use. (John Hickey/Buffalo News)

Paige and Tanner Szczesek are on the front end of the learning curve when it comes to keeping a proper balance. Their father, Shane, also was diagnosed as a child with Type 1 diabetes.

Greater speed is a godsend for the children, each of whom has a continuous glucose monitor and insulin pump to help control their blood sugar. Paige also has a personal diabetes manager, as part of her pump, that helps her family determine when she needs more insulin and how much. Sweet treats are always on hand for times when their blood sugar drops.

When she needs more fast-acting insulin, someone needs to decide when to administer it, then see how it's working. Ashley Szczesek uses a smartphone to keep tabs on blood glucose levels for both children. She teams up with Paiges school nurse and teachers to address shortfalls and spikes. There are phone calls or text messages every time blood sugar readings warrant, as well as reports about when and what the first-grader has eaten.

I can't just send my kid into school and say, I'll see you at the end of the day,' Ashley Szczesek said. I have an alarm set on my phone for when they're high or they're low. On top of that, especially during the night, I'll normally set several alarms to get up and check their blood sugars. If they're low, I go wake them up and give them something to bring up their blood sugar. If they're high enough, I give them some extra insulin. Between us as parents and the children, there's a lot of sleep loss."

Paige started using Fiasp last year after the clinical trial ended. Mastrandrea prescribed it off-label. Her brother started taking it a few weeks ago.

Paiges A1c level has dropped by 1 percent, to about 7 percent, higher than those without diabetes but in a good range for someone Paiges age with the condition.

Fiasp can ease the diabetes burden, but not erase it.

Those without adequate health insurance may be unable to cover higher co-pays or other out-of-pocket costs. Some people have not wanted to switch for that reason, Mastrandrea said.

Meanwhile, researchers continue to pursue a biologic cure, as well as an insulin pump that works with a continuous glucose monitor to deliver insulin on a minute-by-minute basis as needed.

In order to do that really well, Mastrandrea said, you want to have insulins that are faster-acting, absorb better and behave the way my pancreas does. Fiasp is in that category.

Some of Dandonas patients already have the most advanced insulin pump, the Medtronic 670G, though the device can be complicated for most adults to use, let alone children, he said, and still needs agents to more quickly bring blood sugars into the balanced range.

Still, for those in the field and for families like the Szczeseks, recent progress has been nothing short of remarkable.

Early prototypes of closed-looped models like the 670G once weighed two to three times that of adult patients, Dandona said, and now we have a tiny device doing the same thing.

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Breakthrough diabetes insulin drug developed with help from Buffalo - Buffalo News

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Barbara Kay: Will 2020 will be the year of reason in the cancel-culture wars? – National Post

In the hard news business, it is well understood that if it bleeds, it leads. In cultural news, the same principle applies. Not a day goes by that we dont see a story of a panel discussion in jeopardy or cancelled, a controversial film withdrawn, or an academic on the incorrect side of a cultural debate de-platformed. One could be forgiven for assuming that cancel culture reigns supreme in the public forum.

Its gratifying, therefore, to report that a speaking engagement featuring a highly controversial researcher and clinician in the hot-button field of gender dysphoria will take place as planned at McGill University. The cancel-culture mould in this case was not broken by chance. A good strategic plan prevented a predictable brush fire of protest from becoming a conflagration.

Within the Department of Psychiatry at McGill University, the Division of Social and Transcultural Psychiatry (DSTP) pursues and promotes research, training and consultation in the domains of social and cultural psychiatry. Under the rubric of the Culture, Mind and Brain Program, an ongoing sub-division of the DSTP, with links to affiliated faculty in other McGill departments and worldwide, the DSTP is presenting a lecture, titled Children and Adolescents with Gender Dysphoria: Some contemporary research and clinical issues, to be delivered Jan. 23 by Dr. Ken Zucker, professor of psychiatry at the University of Toronto.

The cancel-culture mould in this case was not broken by chance

Dr. Zucker, a pioneer and leading expert in the field of gender dysphoria, is a cancel-culture veteran, and remains a magnet for trans activists ire. In 2015, when he headed up the Gender Identity Clinic at Torontos Centre for Addiction and Mental Health (CAMH), a post hed held for decades, he was targeted for condemnation by trans activists, who accused him of practicing conversion therapy, a false allegation that led to his summary dismissal. Dr. Zucker successfully refuted the charge in a later lawsuit against CAMH, resulting in a payout and retractions, but not in reinstatement. Dr. Zucker does in fact endorse supervised hormone therapy where warranted for adolescents on a case-by-case basis, but is a proponent of watchful waiting and for treating patients holistically.

DSTP Professor Samuel Veissire, organizer of this event, has been keeping me up to date on responses. To be honest, when I received his invitation to the talk and I saw Dr. Zuckers name, I entered it into my calendar with a question mark. I assumed the odds were high that he would be de-platformed by an administration browbeaten by activists. Im happy to be proven wrong.

Thats not to say there was no opposition. Queer McGill issued a warning on its Facebook page, repeating the canard about conversion therapy and advising friends that the talk would be given from a non-trans perspective. Prof. Veissire made a point of meeting with Queer McGill to hear their complaints and concern. Their position was basically no conversation without us at first, but he argued persuasively and respectfully that it is also reasonable for parents to assume their right and to honour their responsibility to be involved in decisions around radical physical changes in their children. This outreach in itself, letting people who object to the talk know that their perspectives are welcome in the conversation, I imagine went some distance in calming potentially roiled waters.

The Facebook pages comments were refreshingly diverse, and maturely considered. One queer woman of colour posted, I understand that this talk isnt for everyone, but I feel like we tend to be quick on condemning what we perceive like an attack, and police each other instead of practicing patience with different levels of understanding or even taking the opportunity to speak our truth.

Prof. Veissire also sent out a call for support on a sex research email list. Other scholars then shared it on Twitter. Responses were intended for both the administration at McGill and the public record, to show people or groups who had asked for the talk to be cancelled that there was widespread support for Dr. Zucker.

The Facebook page's comments were refreshingly diverse

Letters attesting to Dr. Zuckers eminence in his field flowed in from authoritative colleagues, such as Northwestern University psychology professor J. Michael Bailey, Columbia Universitys Developmental Psychoendocrinology Program director Heino F. L. Meyer-Bahlburg, University of Toronto psychiatry professor Ray Blanchard, and Harvard University psychology professor Steven Pinker.

Most touching was an ardent testimonial from Pique Resilience Project, a support group for detransitioned women. They wrote that they themselves would greatly have benefited from the more careful, evidence-based treatment approach that Dr. Zucker uses in his clinical practice. Their email concludes, Perspectives like Dr. Zuckers are critical to ensuring that more individuals dont make the mistake we did.

Most people, Prof. Veissire believes, want to see more brave conversations on difficult topics, but are reluctant to be publicly associated with politically unorthodox views. His private conversations with students have convinced him that when they see evidence that other people they trust are also open to these conversations, peoples fears ease up a bit.

Perhaps, as Prof. Veissire mused, 2020 will be the year of reason in the culture wars spin. Perhaps. Just minutes before filing this column, I was made aware that the Pride Therapy Network of Montreal had sent a letter dated Jan. 20 to Prof. Veissires asking that Dr. Zucker be de-platformed. And on Wednesday McGills Joint Board-Senate Subcommittee on Queer People also wrote the organizers to express disappointment and ask that the event be cancelled. It wont be. A more constructive approach would be for their members to attend the presentation and contribute their perspectives to the discussion in a civil fashion.

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Barbara Kay: Will 2020 will be the year of reason in the cancel-culture wars? - National Post

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Stem Cell Banking Market smart Strategies of the Research and Development Process Dagoretti News – Dagoretti News

Stem cell banking or preservation is a combined process of extraction, processing and storage of stem cells, so that they may be used for treatment of various medical conditions in the future, when required. Stem cells have the amazing power to get transformed into any tissue or organ in the body. In recent days, stem cells are used to treat variety of life-threatening diseases such as blood and bone marrow diseases, blood cancers, and immune disorders among others.

The market of stem cell banking is anticipated to grow with a significant rate in the coming years, owing to factors such as, development of novel technologies for stem cell preservation and processing, and storage; growing awareness on the potential of stem cells for various therapeutic conditions. Moreover, increasing investments in stem cell research is also expected to propel the growth of the stem cell banking market across the globe. On other hand rising burden of major diseases and emerging economies are expected to offer significant growth opportunities for the players operating in stem cell banking market.

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The key players influencing the market are:

Cordlife, ViaCord (A Subsidiary of PerkinElmer), Cryo-Save AG, StemCyte India Therapeutics Pvt. Ltd., Cryo-Cell International, Inc., SMART CELLS PLUS, Vita 34, LifeCell, Global Cord Blood Corporation, CBR Systems, Inc.

This report contains:

The global stem cell banking market is segmented on the basis of source, service type, and application. The source segment includes, placental stem cells (PSCS), dental pulp-derived stem cells (DPSCS), bone marrow-derived stem cells (BMSCS), adipose tissue-derived stem cells (ADSCS), human embryo-derived stem cells (HESCS), and other stem cell sources. Based on service type the market is segmented into, sample processing, sample analysis, sample preservation and storage, sample collection and transportation. Based on application, the market is segmented as, clinical applications, research applications, and personalized banking applications.

Stem Cell Banking Market Global Analysis to 2027 is an expert compiled study which provides a holistic view of the market covering current trends and future scope with respect to product/service, the report also covers competitive analysis to understand the presence of key vendors in the companies by analyzing their product/services, key financial facts, details SWOT analysis and key development in last three years. Further chapter such as industry landscape and competitive landscape provides the reader with recent company level insights covering mergers and acquisitions, joint ventures, collaborations, new product developments/strategies taking place across the ecosystem. The chapters also evaluate the key vendors by mapping all the relevant products and services to exhibit the ranking/ position of top 5 key vendors.

Stem Cell Banking Market is a combination of qualitative as well as quantitative analysis which can be broken down into 40% and 60% respectively. Market estimation and forecasts are presented in the report for the overall global market from 2018 2027, considering 2018 as the base year and 2018 2027 forecast period. Global estimation is further broken down by segments and geographies such as North America, Europe, Asia-Pacific, Middle East & Africa and South America covering major 16 countries across the mentioned regions. The qualitative contents for geographical analysis will cover market trends in each region and country which includes highlights of the key players operating in the respective region/country, PEST analysis of each region which includes political, economic, social and technological factors influencing the growth of the market.

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California man donates part of his liver to Conservative rabbi in Pittsburgh – The Australian Jewish News

Eric Stegers heart is full, although his liver is smaller by 60%.

Steger, a 50-year-old man from Sunnydale, California, affiliated with Chabad, was in Pittsburgh earlier this month fulfilling his dream of donating an entire lobe of his liver to help save the life of another.

The liver recipient, Conservative Rabbi Jeffrey Kurtz-Lendner, 53, said he feels like he has been given a second chance at life.

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Kurtz-Lendner, who relocated to Pittsburgh from Iowa for the purpose of obtaining a transplant at UPMC, had been diagnosed with fatty liver cirrhosis, but the doctors did not know how serious it was until they were in the midst of the transplant.

I could have died before I got put onto a list, said Kurtz-Lendner, who, after the Jan. 7 surgery, is still recuperating but has been discharged from the hospital.

Steger, a math tutor at Foothill College in Northern California, has donated stem cells for a bone marrow transplant and platelets many times, and has been wanting to help save a life with one of his organs for years. He even traveled to Israel to donate a kidney, but was ultimately turned down because he had hypertension.

About a year ago, though, he saw a UPMC commercial airing in California that advertised the fact that it was now performing altruistic liver donations.

I decided to give it a try, said Steger.

He then got in touch with Chaya Lipschutz, an Orthodox woman from Brooklyn who donated a kidney to a stranger in 2005, and since then has made it her work to help others find kidney matches. She receives no money for her services.

Lipschutz had made the shidduch with the kidney patient in Israel for Steger that did not work out, he said.

As fate would have it, Lipschutz did know people who needed a live liver transplant. Steger was medically cleared for the procedure, but the first few people with whom he matched found other donors. Lipschutz then turned to message boards to post that she had an able and willing donor.

Now I was a solution in search of a problem, said Steger.

When Kurtz-Lendners sister in Teaneck, New Jersey, happened to see Lipschutzs post, the match was made.

Post-surgery, both donor and recipient are doing well.

Im feeling very positive, said Kurtz-Lendner, noting that full recovery from the procedure will take about a year. Two weeks ago, I was dying. Now, I have another 30 years.

He, his wife Robin, and his oldest daughter will remain in Pittsburgh for at least six months.

Kurtz-Lendner did not meet Steger until after the surgery, and sees him as an inspiration of a human being. I appreciate what he has done. He just saved my life.

Steger returned to California this week. During his time in Pittsburgh, he received warm hospitality from the citys Jewish community, particularly the Bikur Cholim of Pittsburgh, run by Nina Butler, he said.

Patients and families who come here from out of town always remind us of how special our community is, said Butler. As the Bikur Cholim of Pittsburgh, Im simply organizing the generosity of volunteers to provide the specific support that each patient wants. That started before Jeff or Eric arrived, answering their questions about housing, Shabbat observance and kosher food.

Eric is observant and came unaccompanied, so his housing was complicated because the Family House does not allow patients to stay completely alone, Butler explained. We provided home hospitality, and we also organized volunteers to drop off meals for Eric while his hosts were at work. Most of all, we formed relationships with both patients and Jeffs family so they knew there were Pittsburghers who had their backs.

Robin Kurtz-Lendner said that she and her husband felt so supported, even before we got here. Its been incredible. The whole community has been rallying around us and its really been appreciated.

Donating part of his liver was not easy, Steger acknowledged. Still, he wants to encourage others to consider organ donation.

Im not going to sugarcoat it, he said. It was the hardest thing Ive ever done. It was a year out of my life, one full year when I was thinking about this all the time.

There was a battery of tests, the surgery itself, and now the recovery phase, he said, which all carry physical and emotional risks.

But he is hoping what he did will help generate continued interest in organ donation.

I hope my experience will inspire other people to investigate it for themselves, he said.pjc

Toby Tabachnick can be reached

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Advancells Group & IFC Concluded their 3-Day Workshop on Regenerative Medicine – India Education Diary

New Delhi: On Saturday, January 18th, 2020, the Advancells Group & the International Fertility Center together ended their first workshop Sub-Specialty Training in Application of Regenerative Medicine (S.T.A.R. 2020). The three-day workshop had specialized doctors, medical practitioners, learned scientists of Advancells, the leaders in cell manufacturing & processes and IFC, one of Indias most prestigious Fertility institute who were joined by candidates with MBBS/BAMS/BHMS/BPharma & Masters degree in Life Sciences.

The key-note speaker of the workshop was Dr. Rita Bakshi, founder and chairperson of International Fertility Centre, the oldest fertility clinic and one of the most renowned IVF clinics in India, one of the organizers of the event. Participants also had a privilege to listen to Dr. Sachin Kadam, CTO, Advancells and gain hands-on experience in the preparation of PRP; Liposuction method; and Bone Marrow aspiration. All these techniques were talked about at length and demonstrated in the form of manual & kit-based models to help the candidates gain exposure.

Dr. Punit Prabha, Head of Clinical Research and Dr. Shradha Singh Gautam, Head of Lab Operations at Advancells successfully set the base of stem cell biology for the participants who were experts in gynecology field, stem cell research and pain specialist. With the help of detailed analysis of Application of PRP for Skin rejuvenation; Preparation of Micro-fragmented Adipose Tissue and Nano Fat & SVF (Stromal Vascular Fraction) from Adipose Tissue; and Cell Culturing and Expansion in a Laboratory, applicants understood the application of stem cells in aesthetics, cosmetology, and anti-aging.

Vipul Jain, Founder & CEO of Advancells Group said, Educating young scientists about stem cells is important for us. With this workshop we wanted to discuss and share the challenges and lessons we have learned in our journey of curing our customers. We wanted to establish more concrete knowledge base in the presence of subject matter experts and help our attendees in more possible ways. We are hopeful to have successfully achieved what we claimed with this workshop.

Given the resounding success of the Sub-Specialty Training in Application of Regenerative Medicine (S.T.A.R. 2020), its hoped that the future events shall offer even greater wisdom to the participants by helping them improve and the lead the community into the age of greater awareness.

Advancells Group Advancells is leading the field of stem cell therapies in India and abroad, with representative offices in Bangladesh and Australia. The company provides arrangements for stem cell banking and protocols for partner doctors and hospitals which they can use for treating the patients using regenerative medicine. With a GMP compliant research and processing center that works on different cell lines from various sources such as Bone Marrow, Adipose Tissue, Dental Pulp, Blood, Cord Tissue etc. Advancells also intends to file a patent for this processing technology soon.

For more information, visit

International Fertility Centre IFC is Indias leading fertility center under the leadership and guidance of Dr. Rita Bakshi. She along with her solid team of experienced doctors have create a network of 10+ IVF clinics located in India and Nepal. Their services include In-vitro Fertilization (IVF), Intrauterine Insemination (IUI), Intracytoplasmic Injection (ICSI), Egg Donation, Surrogacy, Blastocyst, Assisted Hatching, Hysteroscopy, Laparoscopy and much more.

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Advancells Group & IFC Concluded their 3-Day Workshop on Regenerative Medicine - India Education Diary

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Cell therapy trialed in mice offers diabetes treatment hope – SelectScience

New cell treatment could help maintain healthy blood sugar levels

A new cell treatment to enhance islet transplantation could help maintain healthy blood sugar levels in Type 1 diabetes without the need for multiple transplants of insulin producing cells or regular insulin injections, research suggests.

In Type 1 diabetes the insulin-producing cells of the pancreas are destroyed. Insulin injections maintain health but blood glucose levels can be difficult to control. Currently in the UK it is estimated that approximately 400,000 people in the UK have type 1 diabetes.

The current recommendation for people with type 1 diabetes who have lost awareness of low blood glucose levels is the transplantation of islets the insulin producing part of the pancreas.

A study in mice found that transplanting a combination of islets with connective tissue cells found in umbilical cords known as stromal cells - could potentially reduce the number of pancreases required for the procedure.

Mice that received the islet-stromal cell combination were found to have better control of blood glucose and less evidence of rejection of islets after seven weeks, compared to those that received islets alone.

In humans, more than two donor pancreases, which are scarce, are often needed because islets can be rejected and are slow to form new blood supplies.

Therefore, multiple islet transplantations and anti-rejection medication are required to control blood sugar levels in people with Type 1 diabetes. Scientists at the University of Edinburgh hope their findings could be a way of overcoming these issues.

The researchers found that islets combined with stromal cells successfully returned normal blood glucose levels just three days after transplantation.

Other studies have used cells sourced from bone marrow and fat. This is the first to use stem cells from umbilical cords and has produced superior results.

The research is published in the journal Science Translational Medicine and funded by Chief Scientist Office in Scotland and Diabetes UK.

Shareen Forbes, Professor of Diabetic Medicine at the University of Edinburgh and Lead Physician for the Islet Transplant Program in Scotland, said: Should this research prove successful in humans, we could reduce the number of islets needed to control blood sugar levels using this co-transplantation approach. This would mean more people with Type 1 diabetes could be treated using islet transplantation while significantly reducing the waiting time on the transplant list.

John Campbell, Professor and Associate Director Tissues, Cells & Advanced Therapeutics at the Scottish National Blood Transfusion Service has said that further work is needed to establish the long-term safety of using this type of stromal cell in this setting before proceeding to clinical trials in humans.

Dr. Elizabeth Robertson, Director of Research at Diabetes UK, said: Islet transplants have been life changing for some people with Type 1 diabetes, treating dangerous hypo unawareness. But there currently arent enough donated pancreases to go around, and the procedure itself isnt yet as effective as it could be.

This new research from the University of Edinburgh is a promising step forward, and one we hope will lead to islet transplants becoming both more effective and more widely available in the future.

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Royal Biologics Announces the Launch of Cryo-Cord, the First Non-DMSO Viable Umbilical Cord Graft – Yahoo Finance

Debuts at the NY20 Foundation for Podiatric Medicine Meeting

HACKENSACK, N.J., Jan. 21, 2020 (GLOBE NEWSWIRE) -- Royal Biologics, an ortho-biologics company specializing in the research and advancement of novel ortho-biologics solutions, today announced the launch of Cryo-Cord, the first DMSO-free viable umbilical cord graft. The company will be showcasing Cryo-Cord along with its new portfolio of Autologous Live Cellular (ALC) technologies at the NY20 Foundation for Podiatric Medicine meeting, held January 24-26 in New York, NY for more than 1500 clinical attendees.

The company will feature its full suite of surgical biologic offerings at exhibit booth #322, and on the podium for Innovation Theater presentations at 10:30am on Friday 1/24/20 and 12pm on Saturday 1/25/20. These scientific presentations will feature several products within the Royal Biologics portfolio. At its booth, Royal Biologics will showcase its comprehensive ALC portfolio designed to personalize live regenerative healing for a wide variety of wound types across the orthopedics continuum.

The launch of Cryo-Cord enables providers with the first DMSO-free viable umbilical cord tissue. Cryo-Cord has been obtained with consent from healthy mothers during cesarean section delivery and is intended for use as a soft tissue barrier or wound dressing. Cryo-Cord is processed using aseptic techniques and frozen with a proprietary cryoprotectant.

Cryo-Cord offers a new enhancement to traditional wound care therapies and we are excited to pave the way with the first DMSO-free cryoprotectant graft on the market, said Salvatore Leo, Chief Executive Officer of Royal Biologics.

Other featured products at NY20 will include Maxx-Cell, which was launched as the world's most advanced bone marrow aspiration device. Maxx-Cell offers a new technique to a gold standard approach of aspirating a patients autologous bone marrow cells. Maxx-Cell however does not require centrifugation to deliver a final end product. The Maxx-Cell system maximizes stem and progenitor cell yields by giving the surgeon the ability to efficiently harvest bone marrow from multiple levels within the medullary space, while restricting dilution of peripheral blood. As a result, Maxx-Cell delivers a high, most pure enriched form of bone marrow aspirate without the need for centrifugation.

This month, the company has also announced the launch of MAGNUS, which is a DMSO-free viable cellular bone allograft and demos will be available during the conference. MAGNUS presents a unique solution to traditional viable cellular allograft technology as it utilizes a DMSO-free cryoprotectant. This novel approach to the viable cellular allograft market differentiates MAGNUS from other technologies currently available.

Leo added, We are excited to participate in NY20 and share how our Autologous Live Cellular based therapies give the surgeons an efficient and effective way to enhance surgical outcomes by providing alternatives to conventional therapies for bone and soft tissue related injuries. We also believe that in a cost-conscious industry, we can provide novel viable cellular products that provide value at the point of care.

To watch the latest ALC product videos and learn more about the range of regenerative medical products offered by Royal Biologics, along with a schedule of 2020 conferences, visit

About Royal Biologics

Royal Biologics is an ortho-biologics company specializing in the research and advancement of Regenerative Cellular Therapy. Its primary focus is on using autologous bioactive cells to help promote healing in a wide range of clinical settings, with its portfolio of FDA-approved medical devices. For more information on its line of products, visit

For more media information, contact:Lisa Hendrickson, LCH Communicationslisa@lchcommunications.com516-767-8390

A photo accompanying this announcement is available at

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BrainStorm Cell Therapeutics COO and CMO, Dr. Ralph Kern, to Present at the 10th Annual California ALS Research Summit – Yahoo Finance

NEW YORK and LOS ANGELES, Jan. 21, 2020 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics Inc. (BCLI), a leading developer of adult stem cell therapeutics for neurodegenerative diseases, announced today that Ralph Kern, MD, MHSc, Chief Operating Officer and Chief Medical Officer, will present at the 10th Annual California ALS Research Summit, January 24-25 at Cedars-Sinai Medical Center, Los Angeles, California.

Dr. Kern will provide an update on BrainStorms Phase 3 ALS Clinical Trial on Friday, January 24, 10:30 -11:10 AM PT, during the session: CIRM funded Stem Cell Clinical Trials in California Updates.

Dr. Kern stated, This prestigious Summit works to increase, expedite and promote the amount and level of amyotrophic lateral sclerosis (ALS) research done in California that has been reinforced and amplified by the international ALS scientific and medical community. I am pleased to have the opportunity to share all that BrainStorm has accomplished in our fully enrolled Phase 3 clinical trial of NurOwn(NCT03280056).

Chaim Lebovits, President and CEO of BrainStorm, stated, California continues to be a global leader in stem cell research and scientific funding. Due to Californias commitment to stem cell scientific investigation, BrainStorm is at an inflection point as we bring our investigational therapy, NurOwn, toward the submission of a biological license application. In July 2017, BrainStorm was awarded a grant of $15.9 million from the California Institute for Regenerative Medicine (CIRM) and three of Californias most prestigious medical centers: University of California, Irvine, Cedars-Sinai Medical Center, and California Pacific Medical Center have contributed immensely to advancement of NurOwn. Everyone at BrainStorm is proud Dr. Kern will have the opportunity to present to the ALS community of California all that has been accomplished due to their ongoing support and encouragement.

About The California ALS Research Summit:

The California ALS Research Summit is the tenth annual gathering of researchers, investigators, clinicians, biotech companies, government representatives, partner organizations, and advocates in ALS and related fields in the State of California.

The purpose of the Summit is to help increase, expedite and promote the amount and level of amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig's Disease) and related research done in California; and to foster networking, collaboration and cooperation among investigators, their peers and their colleagues to identify, develop and deliver new and effective treatments, ideas and, ultimately, cures for ALS.

The result of our efforts is an ongoing roadmap for ALS research in California, which will provide the basis for partnering within the state and other supporters to further studies to find new treatments and ultimately a cure for the disease.

About NurOwn

NurOwn (autologous MSC-NTF cells) represent a promising investigational approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. NurOwn is currently being evaluated in a Phase 3 ALS randomized placebo-controlled trial and in a Phase 2 open-label multicenter trial in Progressive MS.

About BrainStorm Cell Therapeutics Inc.

BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (U.S. FDA) and the European Medicines Agency (EMA) in ALS. BrainStorm has fully enrolled a Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six sites in the U.S., supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. For more information, visit BrainStorm's website at

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Safe-Harbor Statement

Statements in this announcement other than historical data and information, including statements regarding future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could causeBrainStorm Cell Therapeutics Inc.'sactual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorms need to raise additional capital, BrainStorms ability to continue as a going concern, regulatory approval of BrainStorms NurOwn treatment candidate, the success of BrainStorms product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorms NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorms ability to manufacture and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorms ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation,; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available at These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.


Corporate:Uri YablonkaChief Business OfficerBrainStorm Cell Therapeutics Inc.Phone:

Media:Sean LeousWestwicke/ICR PRPhone:

Or:Katie GallagherLaVoieHealthSciencesPhone: + 1 617-374-8800

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BrainStorm Cell Therapeutics COO and CMO, Dr. Ralph Kern, to Present at the 10th Annual California ALS Research Summit - Yahoo Finance

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Darzalex EU nod marks first newly diagnosed MM treatment in six years – PharmaTimes

Janssen has announced that the European Commission (EC) has grantedmarketing authorisationfora new Darzalex (daratumumab) combo, for newly diagnosed, transplant eligible patients with multiple myeloma (MM).

On the news, the combination, which consists of the biologic combined with bortezomib, thalidomide and dexamethasone(VTd) is now the first regimen approved in over six years for newly diagnosed patients who are eligible for a stem cell transplant. It also means that the patient population now has their first opportunity to be treated with a monoclonal antibody.

The company says that the approval was based on results from part one of the Phase III CASSIOPEIA (MMY3006) study, which showed that after consolidation, the stringent complete response (sCR) rate was 9% higher in the Darzalex-VTd arm than the VTd alone arm.

Further, at a median follow-up of 18.8 months, PFS was significantly improved in the Darzalex-VTd group, with the addition of the drug resulting in an 18-month PFS rate of 93%, compared to 85% for VTd alone.

The effectiveness of first line treatment is critical to maximise time until relapse, explained Philippe Moreau, principal investigator and Head of the Haematology Department at the University Hospital of Nantes.

He continued, The CASSIOPEIA study answered that question definitively, demonstrating that the addition of Darzalex in combination with VTd can lead to very deep remissions and also prolong PFS. Im pleased to see the European Commission have recognised this as well.

MM is an incurable blood cancer that starts in the bone marrow and is characterised by an excessive proliferation of plasma cells. In Europe, more than 48,200 people were diagnosed with MM in 2018, with more than 30,800 deaths related to the disease.

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Darzalex EU nod marks first newly diagnosed MM treatment in six years - PharmaTimes

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Controlling Our Own Evolution: What is the Future of Gene-Editing? – The Globe Post

In November 2018, Chinese biophysics researcher He Jiankuimade a historic announcement.

Two twin girls nicknamed Lulu and Nana had become the worlds first genetically modified human beings.

Using a gene-editing technology known as CRISPR, He had manipulated the DNA of the embryos that would become the girls in an effort to make them immune to the HIV virus.

What first seemed like a historic triumph of science, however, quickly became one of the most infamous scandals in medical history.

The researcher was swiftly fired from his university, put under police investigation, and denounced by experts around the world who said he jumped the gun and carried out an experiment that was unsafe and unethical.

In December, He was sentenced to three years in prison for illegally carrying out human embryo gene-editing intended for reproduction. Its unclear whether the experiment caused any genetic damage to Lulu and Nana or if they are even resistant to the HIV virus.

Kiran Musunuru, one of the worlds foremost genetics researchers, was the first expert to publically condemn Hes experiment.

Nonetheless, Musunuru says the birth of the Chinese twins marks the beginning of a new human era, the possibilities of which are boundless.

Potential future implications of gene-editing technology range from preventing genetic diseases to producing designer babies with custom traits to creating superhumans and controlling our own evolution.

With the release of his new book, The CRISPR Generation: The story of the Worls First Gene-Edited Babies, The Globe Posts Bryan Bowmanspoke to Musunuru about where this technology could go from here and what it could mean for the future of humanity.

The following interview is lightly condensed and edited for length and clarity.

Bowman: Could you explain what CRISPR is broadly and how that technology evolved to where it is today?

Musunuru: CRISPR is one type of gene-editing tool. Gene editing is a technology that allows us to make changes to genes in the DNA and in the cells in the body. If were talking about human beings, typically were talking about changes that are related to health or disease.

There are several types of gene editing tools, but CRISPR is by far the most popular one. CRISPR is interesting because it wasnt invented. It actually exists naturally in all sorts of bacteria. It evolved as a sort of an immune system that can fight off viral infections. Just like we can get viral infections, it turns out bacteria can get viral infections as well. And so bacteria created a system by which they can fight off viruses. So thats where CRISPR came from.

Over the past couple of decades, a variety of very talented scientists identified it, discovered it in bacteria, and then were able to adapt it into a gene-editing tool that can now be used in human cells.

What we can do with CRISPR is either turn off genes and thats easier to do or we can make more precise changes to genes such as correcting a mutation that causes disease.

Bowman: Last year, there was the famous or infamous case where Dr. He Jiankui in China covertly created the first gene-edited babies. And I understand that you were the first expert to publicly condemn the experiment. What exactly did Dr. He do and why did you feel it was so unethical?

Musunuru: What he was trying to do was use CRISPR to turn off a gene called CCR5. By turning off this gene, he was hoping to make the babies that were born resistant to HIV infection, HIV being the virus that causes AIDS.

There are many people who are naturally born with this chain turned off and theyre resistant to HIV. So the rationale was, well, Im going to try to create babies who have the same trait.

What he did was problematic for two reasons. One, it was, to put it lightly, a scientific disaster. Everything you worry about going badly with CRISPR actually did happen. Any technology has a potential for a lot of good with the potential for bad. I compare it to fire. It can be very useful. But if youre not careful, it can cause wildfires and a lot of damage and hurt a lot of people. Its the same with CRISPR. It can do a lot of good. It can help patients who have bad diseases. But if youre irresponsible with it, it could actually cause unintended genetic damage.

Its not clear whether these kids that were born they were twin girls nicknamed Lulu and Nana its not clear whether theyre actually protected against HIV infection. Its not clear whether they might have suffered some genetic damage that might have health consequences for them. Its not clear whether the genetic damage if it did occur could get passed down to their children and affect future generations.

So scientifically, there are a lot of problems with it. The work was very premature. I would say that if we were ever going to do this in a reasonable, rational, safe way, were years away from doing it. But he went ahead and just did it anyway. You can call him a rogue scientist, as clich as it is. And he did it in conditions of secrecy. There was essentially no oversight. And potentially these twins and future generations might suffer the consequences.

The other problem is a problem of ethics. The way in which he did it basically violated every principle of ethical medical research in the textbook. Basically, everything that you could do wrong, he did it wrong.

Whenever we do an experimental procedure, we hope that the benefits greatly outweigh the risks. What he was trying to do was protect these kids from HIV. But the truth is, they were in no particular danger of getting HIV compared to the average person. In China, the prevalence of HIV is about 0.1 percent. So there wasnt really much for them to gain. Even if they did somehow during their lifetime get the HIV infection, we have good treatments to prevent it from proceeding to full-blown AIDS.

So what was the benefit of doing this procedure? You have to balance that against the harms. And the genetic damage thats possible that raises risks of things like cancer and heart disease and other diseases. When you have those risks and very little benefit, then its just not a favorable ratio. And thats intrinsically unethical.

Bowman: Seeing as you said that were years away from doing something like this in a more responsible and ethical way, what are the greatest challenges to getting to a point where parents will have the option to go forth with a gene-editing procedure that might prevent their children from suffering from some kind of genetic disease?

Musunuru: There are really two aspects to this. One is a scientific or medical aspect. Can we get to a place where gene-editing of embryos is well-controlled? Where we know that what were doing is truly safe and appropriate from that perspective?

The second issue is really a decision more for broader society. Is this something that we should be doing, something we want to be doing? This is less about the science and more about ethics and morality and legality and religious values and all sorts of other things. Reasonable people can disagree on whats appropriate and whats not appropriate.What complicates things here is that its not really an all or nothing decision. There are different scenarios where you could see parents using gene-editing on behalf of their unborn children.

I like to break it down is three scenarios. The first scenario is with parents who have medical issues that make it so that theres no way they can have natural biological children or healthy babies if they both have a bad disease and theyre going to pass it on to all of their kids unless you do something like editing. These are unusual situations, but they do exist.

The second scenario is one where parents might want to quite understandably reduce the risk of their child having some serious illness at some point in their lifetime. Im talking about things that are fairly common, like Alzheimers disease or breast cancer or heart disease or whatnot. Theres no guarantee that the editing will eliminate that risk. But you can see how parents might want to stack the odds in their kids favor. Its still medical, but its not perhaps as severe a situation with a kid whos definitely going to get the disease unless you do something.

The third scenario would be cases in which parents want to make changes that are not really medical but are more of what we would think of as enhancements. These could be cosmetic changes like hair color, eye color, things like that.

But it could potentially be much more serious things like intelligence or athletic ability or musical talent. Now, to be fair, thats theoretical. I dont think we are anywhere near knowing enough about how genes influence these things to be able to do it anytime soon. You might actually have to change hundreds of genes in order to make those changes. But you can imagine how certain parents might want to do that, might want to advance their children in the ways that they feel personally are desirable.

Bowman: Can gene editing only be performed on embryos or is it possible to edit genes in later stages of pregnancy or even post-birth?

Musunuru: Theres actually a lot of exciting work going on using gene editing to help patients, whether its adults or children. Right now its been focused mostly on adults who have terrible diseases and its really being used as a treatment to alleviate their suffering or potentially cure the diseases.

Just recently, we got the exciting news that two patients one in the U.S. and one in Europe were participating in a clinical trial. They each had a severe blood disorder. One of them had sickle cell disease. The other had a disease called beta-thalassemia. Earlier this year, they got a CRISPR-based treatment. And whats very exciting is that it looks like not only have their conditions improved significantly, it looks like they might actually be cured.

If that bears out, it would really be historic because these are diseases that affect millions of people around the world and were previously incurable. This treatment is also being explored for things ranging from cancer to liver disease to heart disease.

So theres enormous potential for benefit for living people who have serious diseases. But its a very different situation than editing embryos because youre talking about a person who is in front of you. We are trying to alleviate their suffering. That patient has the ability to freely give consent to the procedure, to weigh the benefits and risks and come up with a decision.

Bowman: How does that work? Is it some kind of cell transplant where the new cells then replicate throughout the rest of the body?

Musunuru: Yeah. It depends on the situation. I mentioned those two patients with the blood disorders. The way it worked there was the medical team used bone marrow stem cells. They basically took bone marrow as if they were going to do a transplant and then edited blood stem cells in a dish outside of the body to fix the genetic problem. And then they took those edited stem cells and put them back into the same patient. Those cells start making the blood cells that are now corrected or repaired. And by doing that, to cure the disease.

Another potential implementation is I work on heart disease. And what wed like to be able to do is turn off cholesterol genes in the liver. So what I envision is that a patient with heart disease would get a single treatment and it would deliver CRISPR into the liver and just the liver. It would turn off genes that produce cholesterol in the liver. The effect of that is permanent reduction of cholesterol levels and lifelong protection against heart disease.

This actually works really well in mice. Ive been working on this in my own laboratory for six, almost seven years now experimenting with it in monkeys. And if looks like it works and Im pretty confident that it will work we could be looking at clinical trials in a few years where were taking patients who have really bad heart disease or a very high risk for heart disease and actually giving them the single treatment within their own bodies that would turn off these cholesterol genes.

Bowman: In terms of more cosmetic applications, theres this popular idea that designer babies will be a reality at some point in the future. But how feasible would it be to use gene-editing for something very basic like choosing eye color or hair color? Are there many genes involved in determining traits like that? Are we close to being able to do that if we choose to?

Musunuru: Well, eye color, hair color, those actually turned out to be fairly simple. Theres only a small number of genes that control those. So in theory, if you wanted to do it, it wouldnt be that difficult.

Personally, my point of view is thats a trivial thing. Like why would you go through all that trouble? Do I care if your kid has blue eyes versus green eyes versus brown eyes? Maybe some parents feel that thats very important. So I think simple things like hair color, like eye color, it could be done fairly readily. I just dont see it as serious enough to warrant doing it.

The more complex things like intelligence, gosh, thats going to be so challenging. I mean, intelligence is just such a complex phenomenon. Theres some genetics involved in it, but there are so many other factors that come into that like upbringing and environment. Were not even getting close to an understanding of how someones intelligence comes about, to be perfectly honest about it.

I will point out that even though some of these things are simpler, in general, the vast majority of people are very, very uncomfortable with the idea of using gene editing of embryos for enhancements.

And I think this reflects a couple of things. I think this reflects the fact that people are more sympathetic if something like this is being used for medical purposes and much less comfortable if its being done to give a child an advantage in a way thats not medical.

It brings to mind the recent scandal where wealthy parents were trying to get their kids into good colleges by actively bribing admissions officers, faking test scores, fabricating resums. That kind of thing makes people very uncomfortable that certain people, particularly wealthy people, might try to use this technology to an extreme to advantage their children.

Theres an economic aspect to that. Wealthy parents might have better access to this technology than those who are not as wealthy. And what does that mean? If wealthy parents are somehow able to make designer babies who somehow are advantaged and other people are not, does that exacerbate socio-economic inequalities in our society?

So I think there are a few reasons why people are uncomfortable with the idea of enhancement, whereas on the whole, the majority seem to be at least somewhat open to the idea that there might be good medical uses.

Bowman: Im really happy that you brought up that socio-economic inequality aspect because I was going to ask you about that. But if we table those concerns for a moment and go way out there, theres this notion you write about that we could ultimately, theoretically, control our own evolution.

Ive heard it suggested that it could be theoretically possible to incorporate traits from other organisms that could be advantageous into our own DNA and essentially enter a new post-human stage of evolution. Is that total science fiction or do you think were entering a period where that is increasingly possible?

Musunuru:Well, with the way things are going with this technology. I mean, weve taken a step towards that. But there are many, many, many, many steps that would need to be taken to actually get to that point. But I think youre right. You see the path. We have the technology. Then its a question of perfecting the technology. A question of learning more about what genes from other species might be advantageous.

The cats out of the bag. The technology is here. Whether its five years from now or 10 years from now or 50 years from now or 100 years from now, these sorts of things will inevitably start to happen. And Im not sure theres much that those who would like to not see that happen will be able to do to stop it in the long run.

China Jails Scientist Who Gene-Edited Babies

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Controlling Our Own Evolution: What is the Future of Gene-Editing? - The Globe Post

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What Is Perimenopause And How Young Can It Start? : Shots – Health News – NPR

Katherine Streeter for NPR

Katherine Streeter for NPR

Sarah Edrie says she was about 33 when she started to occasionally get a sudden, hot, prickly feeling that radiated into her neck and face, leaving her flushed and breathless. "Sometimes I would sweat. And my heart would race," she says. The sensations subsided in a few moments and seemed to meet the criteria for a panic attack. But Edrie, who has no personal or family history of anxiety, was baffled.

She told her doctor and her gynecologist about the episodes, along with a few other health concerns she was starting to notice: Her menstrual cycle was becoming irregular, she had trouble falling asleep and staying asleep, and she was getting night sweats. Their response: a shrug.

It wasn't until Edrie went to a fertility clinic at age 39 because she and her partner were having trouble conceiving that she got answers. "They were like, 'Oh, those are hot flashes. It's because you're in perimenopause,' " she says.

If you haven't heard the term "perimenopause," you're not alone. Often when women talk about going through menopause, what they're really talking about is perimenopause, a transitional stage during which the body is preparing to stop ovulating, says Dr. Jennifer Payne, who directs the Women's Mood Disorders Center at Johns Hopkins University.

"Technically, menopause is only one day in a woman's life, which is exactly when she has not had a period for 12 months," she says. "It's the period of time leading up to menopause that causes all the trouble."

And it can start earlier than you might think. Many listeners wrote to us in response to our call-out for individual experiences with menopause to say that they struggled to get medical support for perimenopause in their mid-30s and early 40s.

When Edrie went back to her OB/GYN with the fertility clinic's conclusion, she says the doctor shrugged again and told her that menopause is a normal part of life. She wasn't satisfied with that answer. "Yeah, it's a normal part of life, but it would be great if we could talk about it and figure out strategies."

With that spirit in mind, we reached out to endocrinologists, gynecologists and psychiatrists for advice about navigating this major life transition.

How early can perimenopause start?

It's quite possible for women to start to notice things changing in their mid-30s. Most women arrive at menopause between the ages of 45 and 55, but perimenopause can start as much as a decade beforehand. And about 1% of women in the U.S. reach menopause at age 40 or younger.

How do you know if you're starting perimenopause?

The most telling symptom is changes in your menstrual cycle, says psychiatrist Hadine Joffe, the executive director of the Connors Center for Women's Health and Gender Biology at the Brigham and Women's Hospital in Boston.

"It's the menstrual cycle pattern that really defines this lead-up to menopause," she says. During perimenopause, periods "might be shorter, then a long one, or then a skipped one, or then the flow might be different," says Joffe.

There's no blood or hormone test that can "diagnose" perimenopause. Joffe says a hormone test isn't helpful because hormonal cycles become erratic and unpredictable during this stage.

"There's not really one point in time when a hormone test is done that can be definitive," she says. Even if you took several tests over time, "you might get a very different readout."

Surprisingly, sometimes doctors aren't prepared to help women recognize the start of this life phase. Edrie was upset at her doctors' responses or lack thereof. "I felt so disappointed in the medical industry. How many women has my OB/GYN seen and not recognized the symptoms of perimenopause?"

What symptoms to expect

Be prepared for your PMS symptoms to possibly shift, becoming either more or less extreme, says Dr. Cynthia Stuenkel, a founding member of the North American Menopause Society and a professor and endocrinologist at the University of California, San Diego, School of Medicine. "Women might not get the same kind of breast tenderness or mood shifts that they may have noted in the past," she says.

Mood problems like depression can spike during perimenopause, especially among women who have previously experienced them. Many of our listeners wrote in to say that during perimenopause, they felt incredibly irritable and quick to anger in a way that they had never experienced before.

And of course, many but not all women experience hot flashes, though they may not recognize them. "It's hard, because no one sits us down and teaches us, 'Here's what a hot flash feels like,' " Stuenkel says. "I've seen women who think they're having panic attacks, or heart palpitations. That can be frightening."

Other common symptoms include more frequent urinary tract infections, difficulty sleeping through the night, vaginal dryness that can make sex painful, night sweats and a decrease in libido.

What treatments are there for symptoms?

Some symptoms, like heavy or irregular periods, can be managed with an oral contraceptive, which can "shut down the body's own erratic hormonal fluctuations," says Stuenkel.

"This can kind of be a lifesaver," she says. Such medication may help with hot flashes, too.

In some cases, doctors may prescribe menopausal hormone therapy, or very low doses of hormones to supplement estrogen levels. Stuenkel says it's not a fit for everyone, but it doesn't deserve the bad reputation it has in some circles. She says there was an "exodus" from the use of hormone replacement therapy after the Women's Health Initiative trial halted a study over safety concerns in 2002. But many clinicians now feel much more comfortable using hormone therapy again and usually recommend low doses, selectively, for shorter periods of time.

For people who cannot take estrogen therapy, or choose not to, Stuenkel says some drugs in the antidepressant family, such as SSRIs and SNRIs, can help with hot flashes. Stuenkel says, "While they're not perfect, they can take the edge off and help enough so that women can get a better night's sleep."

There are an abundance of nonhormonal, nondrug treatment options for managing symptoms, some of which have significantly more evidence backing them than others. In 2015, a North American Menopause Society panel found that cognitive behavioral therapy and hypnosis were significantly effective in treating hot flashes. The same panel also found that popular herbal remedies (like black cohosh, dong quai and evening primrose) are "unlikely to help," although some NPR listeners who wrote in said they got relief from some of those treatments.

For depressive and anxiety symptoms, women may want to seek out professional counseling or a psychiatrist.

When do I need to see a doctor?

You might not need to at all. Some people sail right through menopause with little trouble. But if you are experiencing symptoms that are interfering with your life, it's worth making an appointment. Some of these symptoms could indicate other problems that need treatment, such as fibroids or even cancer.

Ways to cope with symptoms

For people approaching this stage of life or who are already going through it, here are four steps for making this transition more manageable.

1. Get educated

"Information is key," says Joffe. She suggests that people approaching perimenopause age empower themselves with knowledge.

The Massachusetts General Hospital Blum Center has a curated list of suggested books. The National Women's Health Information Center has a section on menopause and perimenopause. The American College of Obstetricians and Gynecologists also has a perimenopause FAQ.

2. Monitor your health

Joffe encourages people to track symptoms: "menstrual patterns, hot flash patterns, mood issues, major life triggers." Using a paper calendar or an app to monitor symptoms can make it easier to give your doctor details that can be otherwise hard to remember.

"Knowing that information, somebody can say, 'Well, over the last six months, I only had two periods or I had hot flashes almost every day,' " Joffe says, "or, 'My mood was as bad as it gets for only two days or for a third of the time.' "

And if you bring a thorough health history to your physician and they still give you a shrug, consider a specialist. "There are OB/GYNs that specialize in perimenopause and menopause," Joffe says.

3. Practice smart self-care

Joffe encourages women to protect themselves from things that might worsen their mood or well-being. This includes reducing stress when they can and making sure they get enough sleep.

"Sleep is critical," she says. "Getting a good night's sleep, and making sure it's not broken in the middle of the night."

There are lots of online tools and apps to help with sleep, she adds.

And familiar health advice like getting enough exercise, eating well and moderating alcohol consumption apply to perimenopause too, says Dr. Steven Goldstein who is the co-author of Could It Be ... Perimenopause? and a professor of obstetrics and gynecology at the New York University School of Medicine.

At her doctor's suggestion, Edrie developed a mindfulness practice. She says, "I thought it sounded a little 'woo-woo' at first, but being able to pay attention to what my body is doing and why helps me separate those symptoms from what I need to get through my day. So I'm not overwhelmed by what my body is putting me through."

4. Cultivate community

Most of the women who wrote to NPR about their experiences going through perimenopause said that they felt alone and isolated during this transition.

Having a community to talk to can make it easier to cope with the changes, says Payne, who's going through perimenopause herself. She says she has found support from a few close friends from college.

"To be able to reach out to a group of women who are our same age and say, 'Did you go through this? And, you know, it does provide support. I think that's another version of a coping skill," she says.

Edrie says she joined a few Facebook groups dedicated to perimenopause and found one in particular where she got tips on coping with one of her most troublesome symptoms: brain fog. The conversations made her feel understood and validated.

"I can post about it in this group, and, you know, 10 women will be like, 'Oh, last week, that totally happened to me,' or like, 'I forgot my kid's computer on the top of my car and drove away,' " she says.

She says that being able to commiserate helps her get through symptoms "that maybe don't have a magic pill." Some of her online friendships have even taken shape offline. Edrie has met up with some of the Facebook group members while touring the country with her band.

Now she's a big proponent of finding community and speaking out. "As we get older, we get more and more quiet about what's going on with our bodies and ourselves and our lives. We kind of just, buck up and deal with it."

"And I feel like if we talked more about the things that are happening to our bodies even if we can't actually do anything about some of these things it would just be better for society in general if we were more vocal about it."

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What Is Perimenopause And How Young Can It Start? : Shots - Health News - NPR

Recommendation and review posted by Bethany Smith