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Austin was three years old and Max was a newborn when their mother, Jenn McNary, learned they had a rare genetic condition called Duchenne muscular dystrophy. The doctor painted a grim picture: Her boys would stop walking by age 12 or 13 and, shortly thereafter, they would require nighttime ventilation. They would each need a tracheotomy, a feeding tube, or both by their late teens.

"Our impressive portfolio of life sciences companies underscores the tremendous opportunity for this industry in Central Ohio." COLUMBUS, Ohio (PRWEB) December 17, 2019 Today, Rev1 Ventures, the investor startup studio that combines capital and strategic services to help startups scale and corporations innovate, is announcing the launch of its second fund focused on life sciences companies. The $15MM Rev1 Catalyst Fund II with investment from The Ohio State University, Nationwide Childrens Hospital and Rev1 is three times the size of the first Life Sciences fund and aims to deliver critical investment capital and support for research-based spinouts and healthcare innovators

Intermountain Primary Children's Hospital, along with University of Utah Health, and Intermountain Precision Genomics are teaming up to launch a pediatric center for personalized medicine that will serve the Intermountain West. WHY IT MATTERS The center will use precision genomics to discover, address and treat genetic diseases, many of which affect infants and children and can cause life-long disability. The Center will focus on precision diagnosis, gene therapies and novel therapeutics, and stem cell, immunologic and regenerative medicine.

Three Bangladeshis suffering from a highly debilitating muscular dystrophy, who arrived in Mumbai on Sunday have begun their treatment at a Navi Mumbai spine clinic. Abdus, Rahinul and Shorab aged 24, 14 and 8 respectively were diagnosed with this crippling disease at the time of their birth. They arrived on Sunday evening and we started the treatment on Monday, said Avantika Patil, spokesperson NeuroGen Brain and Spine Institute in Seawoods, Navi Mumbai, who is treating them for free

Prevalence of hypogonadism In the Baltimore Longitudinal Study on Ageing, it was found that 19% of men over 60 years had low testosterone. The Hypogonadism in Males (HIM) study estimated the overall prevalence of hypogonadism at approximately 39% in men aged 45 years or older (18). It has been estimated that only 535% of hypogonadal males actually receive treatment for their condition (19,20).

India Stem Cell Market to surpass huge revenue of USD 1.27 Billion at CAGR +13% by 2028. Stem cell therapy in India helps in treating several diseases, including leukaemia, lymphoma, thalassemia, Parkinsons, Alzheimers, stroke, cerebral palsy, spinal cord injury, muscular dystrophy, etc

Thesis CRISPR Therapeutics (CRSP) is a high-risk, high-reward investment prospect. In my view, the market has mispriced the probability of CRISPR Therapeutics successfully utilizing the CRISPR/CAS9 platform across multiple disease states.

Today's early morning highlights from the major news organizations. Kaiser Health News:Its About Love And Solidarity: Mutual Aid Unites NYC Neighbors Facing COVIDNancy Perez, a 45-year-old resident of the Brooklyn neighborhood of Bedford-Stuyvesant, contracted COVID-19 in March

-Beta thalassemia: Two patients are transfusion independent at 5 and 15 months after CTX001 infusion; data demonstrate clinical proof-of-concept for CTX001 in transfusion-dependent beta thalassemia- -Sickle cell disease: Patient is free of vaso-occlusive crises at 9 months after CTX001 infusion- -Five patients with beta thalassemia and two patients with sickle cell disease have been treated to date with CTX001 and all have successfully engrafted- ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, June 12, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced new clinical data for CTX001, an investigational CRISPR/Cas9 gene-editing therapy, from the CLIMB-111 and CLIMB-121 Phase 1/2 trials in transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD), and highlighted recent progress in the CTX001 development program. These data were presented during an oral presentation at the European Hematology Association (EHA) virtual congress by Dr.

Personalized medicine has taken a big step forward with the launch of non-profit n-Lorem Foundation, which will create patient-tailored antisense oligonucleotide (ASO) therapeutics for people with rare diseases at no cost to the patients. This comes at the same time as custom gene therapies for rare disease patients are being developed, including some combined with CRISPR

Austin was three years old and Max was a newborn when their mother, Jenn McNary, learned they had a rare genetic condition called Duchenne muscular dystrophy. The doctor painted a grim picture: Her boys would stop walking by age 12 or 13 and, shortly thereafter, they would require nighttime ventilation

UCLA Broad Stem Cell Research Center/Nature Medicine At left, image shows white blood cells (red) from one of the X-CGD clinical trial participants before gene therapy. At right, after gene therapy, white blood cells from the same patient show the presence of the chemicals (blue) needed to attack and destroy bacteria and fungus.

Abstract Leber congenital amaurosis (LCA), one of the leading causes of childhood-onset blindness, is caused by autosomal recessive mutations in several genes including RPE65. In this study, we performed CRISPR-Cas9mediated therapeutic correction of a disease-associated nonsense mutation in Rpe65 in rd12 mice, a model of human LCA

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CRISPR.

Gene therapy is the therapeutic delivery of nucleic acid polymers into a patient's cells as a drug to treat disease.[1] The first attempt at modifying human DNA was performed in 1980 by Martin Cline, but the first successful and approved[by whom?] nuclear gene transfer in humans was performed in May 1989.[2] The first therapeutic use of gene transfer as well as the first direct insertion of human DNA into the nuclear genome was performed by French Anderson in a trial starting in September 1990. Between 1989 and February 2016, over 2,300 clinical trials had been conducted, more than half of them in phase I.[3] It should be noted that not all medical procedures that introduce alterations to a patient's genetic makeup can be considered gene therapy. Bone marrow transplantation and organ transplants in general have been found to introduce foreign DNA into patients.[4] Gene therapy is defined by the precision of the procedure and the intention of direct therapeutic effects.

This article is about the medical therapy.

2009 "A UK and Canadian team have manipulated human skin cells to act like embryonic stem cells without using viruses making them safer for use in humans. "Study leader Dr

_______ GENE THERAPY: ______ ______ Caveat: Medicine is an ever-changing science.