Archive for September, 2019

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NORFOLK, Va. -Kaeli Prices piercing cry is a joyful noise to her mom and dad.

She'll be screaming right in your face and all we can think is that we're just so thankful that we can hear her now, said Kelli Price, Kaelis mother.

When Kaeli was born in March, you could barely hear her cry and more signs signaled something wasnt right.

We started noticing she wasn't holding her head up like some babies would normally do, said her mother. So we started to worry a little bit.

Their pediatrician in Belvidere, North Carolina began to worry, too. He sent them to see Dr. Crystal Proud, a pediatric neurologist at Childrens Hospital of The Kings Daughters. The doctor specializes in Spinal Muscular Atrophy. Its the top genetic killer of infants.

The babies that were diagnosed with this disorder passed away from losing the ability to breathe because those muscles became so weak, explained Dr. Proud.

The doctor said the source of the disorder is a missing or mutated gene which keeps the babies muscles from developing. Most diagnosed with SMA die by the age of two. Ten babies a year are diagnosed with the deadly condition in Virginia. At just eight weeks old, testing confirmed Kaeli Price was one of them.

We were overwhelmed, said Kaelis mother.

We felt powerless, said Brandon Price, Kaelis father.

Dr. Proud started Kaeli on a drug called Spinraza, which has shown promise in babies with SMA since it was approved by the FDA in 2016. It requires an injection into their tiny bodies every four months for the rest of their lives.

Not long after Kaelis initial treatment on Spinraza, a newly approved gene therapy drug called Zolgensma hit the market. It would essentially give Kaelis body the missing gene, and it would only require one dose. However, the drug came with a price tag of $2.1 million. The Price familys insurance covered it, allowing Kaeli to be the first baby in Virginia to be treated with Zolgensma under FDA approval.

I can't even describe how thankful we are, said Mrs. Price.

I hope and I expect that she will sit, stand, and walk. She will go through school and just excel, said Dr. Proud. And at some point well down the road she will have her own family that she can tell the story to.

Zolgensma has come under scrutiny because of its multi-million dollar price tag.

Dr. Proud, who was part of the clinical trials for the gene therapy drug, said it is actually cost effective when you weigh the billions of dollars it takes for clinical trials and the medical bills a child would incur without the drug.

Dr. Proud said beginning this year, all newborns in Virginia will be tested for SMA. The earlier doctors can intervene, preferably within the first few weeks of life, the better their chances for treatment.

Link:
Gene therapy drug priced at $2 million saves North Carolina babys life - WTKR News 3

Here's What Happened to Dr. Sharpe on 'New Amsterdam' Details!AcceptBrowsers may block some cookies by default. Click accept to allow advertising partners to use cookies and serve more relevant ads. Visit our privacy policy page for more information.Source: Karolina Wojtasik/NBCBy Amber Garrett

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The good news for New Amsterdam fans who love Helen is that the oncologist, portrayed by Freema Agyeman, survived her injuries in the horrific ambulance accident that ended Season 1. However, there seems to be a significant obstacle jeopardizing her future at the hospital, in the form of a former rival.

The three-month time jump revealed medical director Max Goodwin has been placed under a new doctor's care, Dr. Valentina Castro. When we saw her occupying Helen's office, we felt a pit in our stomachs.

While showrunner David Schulner says it was always going to be Georgia who would not make it, the fervor of "Sharpwin" shippers almost changed that outcome.

"We have a huge fan base who all they want is to see Max and Sharpe together," he said in an interview with TVLine. He says that's "not in the cards now," and since Georgia's death would seem to open that possibility up, he had reservations.

"We don't want them to be together," he continued. "They're the best of friends, and that's the relationship that we're really excited to explore: two adult friends who love and care for each other."

While it's unlikely they would have killed off Helen instead, a change of plans may have affected Lauren Bloom's fate or lessened the impact of the finale by having none of the three women die.

While Helen's still around, she seems to be back in her role as ambassador for the hospital, promoting the revolutionary gene therapy treatment that seems to have Max on a path to being completely cured of his cancer. While that outcome obviously is fantastic news for everyone, including Helen, it does reveal a troublesome rivalry.

Doctors Castro and Sharpe clearly have a history, as it's revealed Helen once fired Valentina. Now that the latter's promising gene therapy research seems vindicated, she's using it to bargain for essentially co-running Helen's department. While Dr. Sharpe ultimately agrees to that arrangement, I doubt that will clean up all the bad blood between these two.

There also seems to be a clear rift between Max and Helen after the accident, and not just because they've removed the complicated doctor-patient dynamic of their relationship. It's too soon to tell whether Max's coldness is a product of his grief or something larger.

However, it's clear from the showrunner's comments about the importance of Max and Helen's friendship that there's no danger of the latter being written out of the story anytime soon even if the nature of that relationship won't satisfy Sharpwin shippers!

New Amsterdam airs Tuesdays at 10 p.m. ET on NBC.

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Here's What Happened to Dr. Sharpe on 'New Amsterdam' Details! - Distractify

When Lizzie Acevedos fraternal twin sons, now 15, were newly diagnosed with autism, she was hopeful about diets and vitamin injections that were being touted as miracle treatments or even cures.

Around age 4, the boys were on a gluten-free, casein-free diet, and for a couple months they mainly ate a special organic brand of chicken nuggets. Wheat products or anything else with gluten were out, as were dairy foods containing the milk protein casein. Acevedo also started giving vitamin B12 injections, prescribed by a Defeat Autism Now! doctor, to her son Omar, who is nonverbal and has more significant intellectual disabilities than his brother, Jorge, who speaks and is more independent.

Back then, I just needed something to make things better, Acevedo said. She tried the treatments for a few months but stopped when she didnt see a noticeable effect.

Now, a decade later, Acevedo has heard about lots of hyped alternative approaches for autism, most recently worrisome reports of parents giving their kids bleach drinks or enemas. However, shes also learned there arent any quick remedies for autism, which affects brain development and is characterized by difficulties with communication and social skills and by restricted interests and repetitive behaviors.

Theres no cure for autism and anybody who tries to sell you a cure is lying, Acevedo, a single parent and a fifth grade teacher in Los Angeles, said.

But she understands why parents of autistic children can fall prey to scams. Ive been where they are now, and I know how desperate it feels to want to get your child to be better, she said.

When autism research started to really accelerate a couple decades ago, many scientists thought finding a cure might be easier. Today, the latest science points away from a single cure, but there are ways to help autistic people lead healthier, happier lives and more that can be done to help.

I think that given the complexity and the variability of the causes and the manifestations of autism, trying to come up with a cure is probably not the right approach, said autism researcher and psychologist Len Abbeduto, director of the University of California, Davis, MIND Institute in Sacramento.

An estimated 80 percent of autism cases involve genetic factors, and it tends to run in families, but there is no single autism gene, Abbeduto explained. In fact, research has shown that more than 100 genes, and maybe upwards of 1,000, may play a role. Researchers also suspect that environmental factors such as exposures to infectious agents, pesticides or other toxins in pregnancy may play a role.

Scientists are investing a lot of work into understanding the genes but were also realizing its a lot more complicated than anybody ever thought when they started out, psychologist Ann Wagner, national autism coordinator for the U.S. Department of Health and Human Services, said.

Its highly likely that there are different causes for different kinds of ASD.

We do know that its highly genetic, we just havent identified how particular kinds of genes might interact with each other or with other factors to cause autism spectrum disorder, Wagner said. Autism is such a heterogenous disorder, so its highly likely that there are different causes for different kinds of ASD.

These research developments come amid growing controversy over whether autism even needs a cure. Autism Speaks, an advocacy and research group founded in 2005, removed the word cure from its mission statement in 2016.

In the beginning, [researchers] were looking more for the magic bullet, the magic pill. We were looking for the autism gene, and we thought that would ultimately lead to some kind of cure of autism, psychologist Thomas Frazier, chief science officer at Autism Speaks in New York, said. Then we recognized that we were way off base.

Now, researchers have turned much of their attention to identifying autism in children as early as possible in hopes of intervening sooner with therapies to try to alter the developmental trajectory of their young brains. While skilled practitioners can diagnose autism in toddlers at 18 to 24 months of age with some research indicating there are detectable signs in babies as young as 6 months most kids arent diagnosed until age 4.

Katarzyna Chawarska, a professor of child psychiatry who leads Yale Universitys Autism Center of Excellence in New Haven, Connecticut, is studying signs of autism in babies. The reason why we are focusing so much on early diagnosis is that it is our hope that by intervening early, we can capitalize on still tremendous brain plasticity that is present in the first, second, third year of life, she said.

The goal, Chawarska said, is to help alleviate the symptoms and make sure that every child with autism reaches their full potential.

If youre trying to get rid of autism, youre trying to get rid of us.

Doctors, for instance, would like to minimize any intellectual disabilities and help patients communicate better and improve socials skills. They also want to quickly identify and address any medical conditions that often accompany autism, such as seizures, gastrointestinal problems, sleep disorders, Attention Deficit Hyperactivity Disorder and anxiety.

Researchers already are seeing positive results with interventions such as behavioral treatments and speech therapy in toddlers.

One of the things that we do know is that intensive early intervention improves outcomes for kids, so the earlier we can intervene the better, Abbeduto said.

The idea of curing autism also has become highly controversial with the growth of the neurodiversity movement, which emphasizes respecting and valuing all people for who they are, regardless of whether they are neurotypical.

The C word raises a lot of attention in the community at large, said Michael Maloney, executive director of the Organization for Autism Research, a group in Arlington, Virginia, that funds research to improve the daily lives of autistic people. The largest objection is from people with autism who see themselves as independent and competent and dont see themselves as broken and needing to be fixed.

Among the critics is Julia Bascom, executive director of the Autistic Self Advocacy Network, a Washington, D.C.-based group that is run by autistic people, including herself.

Self-advocates have been largely successful at saying this concept of a cure is really offensive, she said. Who we are is OK, we just need support.

Bascom isnt opposed to research and therapies that help autistic people as long as they arent trying to strip them of their autistic traits.

If youre trying to get rid of autism, youre trying to get rid of us, and thats something our community takes really personally, she said. There are certainly a lot of co-occurring conditions like epilepsy that a lot of us have that wed like to not have. But we dont tend to feel that way about autism and we get really concerned when we see all this money going into risk factors and causation and genetics as opposed to finding out why autistic people tend to have shorter lifespans, or why our suicide rate is nine times higher than average, or what autism really looks like in adults.

Some of her other questions include why girls and people of color are diagnosed later in life, why autism has so many co-occurring conditions, why people with autism tend to react differently to medications, and why they engage in self-injurious behaviors, such as head banging and skin scratching.

Like Acevedos boys, a growing number of teens and adults are living on the autism spectrum, but Bascom and others say there is far too little research on understanding how autistic people are affected across their lifespan and how to help them live life to the fullest. Most autism research dollars in the United States go toward understanding the biological underpinnings of autism in order to diagnose and treat young children.

Autism research spending in the U.S. totaled more than $364.4 million in 2016, the latest year for which figures are available, with 80 percent of that money coming from federal agencies and 20 percent from private organizations. Of the spending, just 2 percent went toward autism lifespan issues and 5 percent toward services, according to the governments Interagency Autism Coordinating Committee. An additional 35 percent went to biology, 24 percent to risk factors, 16 percent to treatment and interventions, 10 percent to infrastructure and surveillance, and 8 percent to screening and diagnosis.

Paul Shattuck, director of the Life Course Outcomes Research Program at the A.J. Drexel Autism Institute in Philadelphia, and a member of the scientific council of the Organization for Autism Research, agrees that not enough attention is paid to adults with autism.

Were expending a lot of effort for very young children with autism, but as a society we kind of drop the ball once these young people become young adults, he said. Theres really not much there for autistic adults or their families in terms of services or even thinking how to support autistic people across the lifespan.

There arent exact figures on the total number of Americans with autism but by one estimation 3.5 million people are on the spectrum, and diagnoses have been increasing. About 1 in 59 children are on the autism spectrum, according to the latest Centers for Disease Control and Prevention figures from 2014, up from 1 in 150 kids in 2000. While some of the increased prevalence may be a true increase in autism cases, the CDC says that a broader definition of the autism spectrum and improved diagnosis efforts likely contributed to the higher numbers.

By Shattucks latest research estimates, 70,000 to 80,000 or more autistic youths per year will turn 18. Thats close to a million people over the next decade, he said, highlighting an urgent need for research to address the health and well-being of autistic adults.

Autistic kids are eligible for special education services while they are in school, and services can last until age 21, but help is harder to come by after that. When teens exit high school, they fall off what is called the services cliff, Shattuck said. It becomes much more difficult to find help and services once kids age out of eligibility for special education. And the young adult outcomes and the adult outcomes are pretty dismal frankly.

After high school, most young autistic adults do not have jobs, career training or additional educational opportunities. Autistic adults also struggle to find independent living arrangements, maintain friendships, get involved with community activities or have enough money to pay for their needs, said Shattuck, whose center helps autistic people and their families with paperwork for Medicaid, Social Security, group housing and more. Many autistic adults continue to live with their parents, raising concerns about what happens when the parents pass away.

Wagner, the national autism coordinator, agrees there needs to be more research on autism across the lifespan and said the government is trying to attract and fund more research in this area.

Just like parents everywhere, Acevedo wants the best for her kids. But after Omar and Jorge finish high school and special education services end, she wonders and worries about what the future holds.

I would love to see some more money put into the transition of young adults with autism into the most independent living situation they can get, Acevedo said. I would love to see money put into job training, taking the skills that these children have because everybody has skills, something that they can do and just really refining it and making these kids marketable to where they can earn some sort of income. Theres something about getting a paycheck and having your name on it as an adult that means so much, and Im sure its going to mean a lot to my kids.

Shattuck says helping autistic adults or those with disabilities ultimately helps everyone.

Our organizations and our communities function better when we make space for everyone of all abilities, he said. Its about helping ourselves and helping our communities be better, higher quality places for all of us.

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Why the focus of autism research is shifting away from searching for a 'cure' - NBCNews.com

Cancer is a devastating illness. Mostly, because of how rapidly it spreads to other areas in the body. Because of this danger, researchers are developing treatments that have the ability to specifically target abnormal cells and then slow or stop those cells from growing and spreading. This method is known as targeted therapy. As scientists discover more about gene and protein changes, their focus is to develop new drugs that hone in and target those changes.

Targeted therapies offer an alternative treatment option to chemotherapy and radiationwith less severe side effects. The reason why there are fewer side effects is because targeted treatment specifically targets abnormal cells, versus chemotherapy and radiation, which cant distinguish between abnormal and healthy cells.

Malignant mesothelioma is an aggressive, fatal disease that has seen rising cases over the past 60 years. A person with mesothelioma cancer is generally experiencing tumor growth in the lining (mesothelium) of the lungs, abdomen, or heart (pleura, peritoneum, or pericardium). Within the past few years, researchers have been testing the use of targeted therapies on mesothelioma patients in clinical trials.

There are two primary types of targeted therapies: monoclonal antibodies and molecule medicines. Molecule medicines are small enough to dive into cancer cells and obliterate them, and monoclonal antibodies fight cancer cells on the surface, or surrounding areas, and are too big to slip into cancer cells.

Sometimes, oncologists use monoclonal antibodies to launch chemotherapy and radiation treatments straight into tumors. This can occur through an IV in a vein, or as a shot. Monoclonal antibody medication treatments usually end with the stem -mab. Three monoclonal antibody types are:

One of the most common targeted therapies, signal transduction inhibitors block signals that tell cancer cells to divide too much and too fast.

This form of treatment blocks the growth of blood vessels that cancer cells form in order to retrieve nutrients and oxygen. In the case of mesothelioma and some other cancers, the target is a substance called vascular endothelial growth factor (VEGF) and utilizes the drug bevacizumab. Its usually administered in conjunction with chemotherapy medications pemetrexed and cisplatin.

This treatment utilizes the patients immune system to locate and destroy cancer cells. An oncologist can administer immunotherapies that boost the immune system to better attack cancer, or immunotherapies that highlight tumor cells so theyre quicker and easier for the immune system to find and attack.

This treatment is helpful in that it can penetrate cell membranes and interact with targets from the inside of a cell. Molecule medicines are designed to interrupt the enzymatic activity of a specified protein and generally end with the stem -ib. Three molecule medicine variations include:

Another form of targeted therapy, gene expression modulators modify the proteins that control the abnormal instruction or expression of genes in cancer cells.

When cells die after growing old or becoming damaged, this is called apoptosis. Cancer cells tend to avoid this natural process, and apoptosis inducers cause abnormal cells to go through normal cell death.

As with most cancer treatments, targeted treatments come with side effects. A patients experience with these side-effects varies, as each individuals case is unique. More common side effects include:

Rarer, but possible side effects of targeted treatments can include:

Your doctor may have some medications available to help relieve side effects, and its recommended you call or stop by their office if youre experiencing them.

A doctor will need to test your tumors to find targets the treatments can focus on. They may use a biopsy to take these tests, which consists of sampling the tumor and then checking it in a lab. Two patients with the same type of mesothelioma may not have the same targets, and some medications may be ineffective if you do not have certain gene mutations. An oncologist may have to administer other mesothelioma medications or treatments before targeted therapy can be applied.

You dont have to go through this alone. Your oncologist will have resources available if you have any questions or issues during any of your mesothelioma treatment. If targeted therapy sounds like an option for you, definitely run that by your cancer-care team, so they can fully assess whether thats the best option based on your health, mesothelioma stage, and other factors.

A mesothelioma diagnosis is a trying time for patients. Download our free guide to learn more about the disease.

Jennifer Verta thrives as a digital content writer and SEO specialist at Mesothelioma Hub. She has been producing content for clients since before she graduated from the University of Colorado at Denver with a Bachelor of Arts in Communication and a Minor in English Writing. Jens mission is to help promote awareness of mesothelioma to as many people as possible by providing only the most up-to-date and accurate content available. When she isnt cranking the gears at work, Jen can be found snowboarding, hiking, catching live music or socializing with friends.

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Targeted Therapy | Treating Mesothelioma - Mesothelioma Hub

Heart cells derived from patients stem cells and grown in a lab dish can reveal important clues about the development of heart ailments associated with Fabry disease.

The study, A Human Stem Cell Model of Fabry Disease Implicates LIMP-2 Accumulation in Cardiomyocyte Pathology, was published in Stem Cell Reports.

Fabry is a rare genetic disorder caused by mutations in the GLA gene. Located on the X chromosome, the gene provides instructions for the production of an enzyme called alpha-galactosidase A (alpha-GAL A).

These mutations typically affect the activity of alpha-GAL A, leading to the accumulation of a type of fat called globotriaosylceramide (GL-3) in different tissues and organs, including the heart, kidneys and nervous system, gradually compromising their normal function.

For this reason, most Fabry patients develop heart disease over the course of their lives, which may progress to heart failure, the most common cause of death among people living with the disorder.

A major obstacle for advancing therapy for patients with [Fabry disease] is the knowledge gap between the direct molecular consequences of alpha-GAL A deficiency in CMs [cardiomyocytes, or heart cells] and the cascade of events driving disease in the heart; the inaccessibility of CMs from patients precludes adequate investigation of these events, especially at early stages, the investigators wrote.

In a previous study, researchers describe the generation of induced pluripotent stem cells (iPSCs) from Fabry patients carrying nonsense mutations in the GLA gene. This gave them the possibility, for the first time, to study the impact of alpha-GAL A deficiency on heart cells derived from patients iPSCs grown in a lab dish.

(iPSCs are fully matured cells that are reprogrammed back to a stem cell state, where they are able to grow into any type of cell. A nonsense mutation is a mutation in which the alteration of a single nucleotide (the building blocks of DNA) makes proteins shorter.)

Investigators from Sanofi, in collaboration with researchers at the University of Manchester, further investigated the properties of heart cells derived from patients iPSCs. Their aim was to discover more clues about the molecular mechanisms involved in the development of heart disease linked to Fabry.

Functional and structural characterization experiments revealed that heart cells from Fabry patients had higher levels of GL-3, and showed a series of abnormalities in the way they responded to electrical stimuli and in how they regulated their calcium usage, compared to heart cells from healthy people serving as controls. Calcium is essential to coordinate the hearts function by contributing to the electrical signals involved in heart muscle contraction.

When researchers analyzed the protein contents of heart cells grown in a lab dish, they found these cells produced more than 5,500 different proteins. This analysis also showed that compared to controls, heart cells from Fabry patients produced large amounts of lysosomal membrane protein 2 (LIMP-2) and heat shock-related 70 kDa protein 2 (HSPA2/HSP70-2).

(LIMP-2 is a protein normally found on the membrane of lysosomes small structures within cells that accumulate, digest, and recycle materials that regulates their transport within cells; HSPA2/HSP70-2 is a protein involved in cellular quality control, participating in the folding of other proteins and targeting abnormal proteins for degradation.)

Heart cells from Fabry patients released high amounts of cathepsin F, a protein that helps breakdown materials being transported inside lysosomes, as well as HSPA2/HSP70-2. As expected, when researchers corrected the genetic mutation associated with Fabry in heart cells derived from patients iPSCs, all these defects were reversed.

To confirm the validity of these proteins as Fabry biomarkers, researchers then forced healthy heart cells to produce high amounts of LIMP-2. They discovered this also triggered the release of large amounts of cathepsin F and HSPA2/HSP70-2, resulting in a massive accumulation of vacuoles (enclosed compartments filled with water and other substances) inside cells.

In summary, our study has shown the power of the iPSC model to reveal early functional changes and the development of a distinctive biomarker expression profile in [Fabry disease] CMs. These biomarkers may be of utility in drug screening and in elucidating the earliest pathological events and cascades in [Fabry disease] cells. Quantification in patient plasma and urine samples will be an important next step toward validating their relevance in patients, the researchers wrote.

A better understanding of these mechanisms will no doubt accelerate the development of more effective and increasingly personalized therapies for patients, they added.

Joana is currently completing her PhD in Biomedicine and Clinical Research at Universidade de Lisboa. She also holds a BSc in Biology and an MSc in Evolutionary and Developmental Biology from Universidade de Lisboa. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells cells that make up the lining of blood vessels found in the umbilical cord of newborns.

Read more:
Fabry Heart Cells Grown in Lab Dish Give Hints to Cardiac Complications - Fabry Disease News

Artificial intelligence has been embraced for its ability to offer insight from big data. By applying the technology to genetics, a research team led by Queen Mary University of London has found clues that they say could aid the development of new drugs for heart failure and identify people at risk of the disease.

Based on an AI analysis of heart MRI images from 17,000 volunteers in UK Biobank, the researchers linked genetic factors to 22% to 39% of abnormalities in the size and function of the hearts left ventricle, which pumps blood into the aorta. They published the findings in the journal Circulation.

The team identified or confirmed 14 regions in the human genome that play a part in determining the size and function of the left ventricle, becausethey contain genes that regulate the early development of heart chambers and the contraction of heart muscle. Enlargement of left ventricle is a condition that can hamper the heart muscles ability to contract and pump blood, putting the patient at high risk of heart attack.

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This study has shown that several genes known to be important in heart failure also appear to regulate the heart size and function in healthy people, said study co-author Steffen Petersen of Queen Mary in a statement. That understanding of the genetic basis of heart structure and function in the general population improves our knowledge of how heart failure evolves.

RELATED:Bayer teams up with AI firm Sensyne Health to mine NHS data for its heart disease pipeline

There is a growing interest in using AI to gain insights into cardiovascular disease. Bayer recently partnered up with Sensyne Health, which uses AI to mine patient data from the U.K. National Health Service, including genomic sequencing data and real-world evidence, to help design clinical studies and accelerate drug discovery.

Many research teams having been looking at different ways to treat heart disease, including using immune therapies and regenerative approaches. Scientists at the University of Pennsylvania, for example,developed genetically modified T cells to attack and remove cardiac fibroblasts, which can lead to cardiac fibrosis. Vanderbilt University researchers identified Roches SYN0012, originally designed to treat rheumatoid arthritis, as a promising candidate that could dampen inflammation of heart tissue after a heart attack. Such inflammation can progress to acute episodes andchronic heart failure.

To help repair damaged cardiac tissue after a heart attack, scientists at the University of Cambridge in the United Kingdom and the University of Washington combined two types of cells derived from human stem cellsheart muscle cells and supportive epicardial cells that help the muscle cells live longer. A team at the the Morgridge Institute for Research previously added a drug called RepSox to stem cells to build better smooth muscle cells that can grow into functional arterial cells.

The Queen Mary researchers believe the 14 regions of the genome they fingered in their new study could be just the beginning of a larger story about genes and heart disease. Our academic and commercial partners are further developing these AI algorithms to analyze other aspects of cardiac structure and function,lead researcher Nay Aung said in the statement.

Aung and colleagues argue the genetic markers theyve already uncovered could help identify those at high risk of developing heart disease or open up new avenues for targeted treatments. The genetic risk scores established from this study could be tested in future studies to create an integrated and personalized risk assessment tool for heart failure, Aung said.

Link:
AI uncovers genes linked to heart failure - FierceBiotech

All living organisms function under specific conditions, the so-called laws of nature. But with the expansion of scientific knowledge in the past centuries, humans havent always just been helplessly swayed by the powers of nature.

While far from controlling nature, we have invented substances and came up with strategies that allow us to stretch the boundaries of science, from altering cell processes to becoming fully immune to diseases.

One of the most promising innovations of our times nanotechnology is taking it even further. Sometimes nicknamed the next industrial revolution, it pervades virtually every field from manufacturing to the food and drink industry.

By operating on the atomic and molecular scale, nanotechnology carries out the most precise interventions. What new possibilities does it bring to industries dealing with biology?

In 2018, global healthcare nanotechnology reached $160 billion and this number is likely to grow to over $300 billion by the end of 2025. Theres no wonder why healthcare is the true harvest field of nanotechnology with diverse opportunities, mostly in diagnostics, treatment, and prevention of diseases itself.

Experts have developed nanosized diagnostic devices that can be deployed throughout the human body to monitor levels of toxins or other substances. This allows for constant and real-time monitoring of an individual on a very detailed level something that was hardly imaginable just a few years ago.

Read More:Challenges in achieving precision medicine, personalized healthcare

And whats more! due to their size, these tiny sensors can enter spaces that are normally difficult to examine, including the brain.

Regarding treatment, there have been experiments with nanosized robots that can travel through bodily fluids. They can work to deliver active substances in a highly effective way.

Likewise, they could bring implants that destroy old cells and inject healing substances to promote the growth of new ones or the recovery of existing ones.

In this respect. In fact, researchers at North Carolina State University are developing a method to deliver cardiac stem cells to damaged heart tissue.

This highly-targeted approach also means a revolution in cancer treatment as we know it: nanotechnology could eliminate the adversary side effects of the conventional methods that affect the whole system.

Read More:Doctors will navigate this passive pill cam like they were playing Xbox

And with closer oversight and enhanced possibilities for direct intervention, cancerous cells can be destroyed even before a major breakout occurs.

Scientists at the Worcester Polytechnic Institute are working on such non-invasive preventive strategies. They have developed a chip made of carbon nanotubes that can capture circulating tumor cells of all sizes.

These can be analyzed easily to help identify any early-stage tumors and monitor treatment progress.

Nanotechnology has also seen a big boom in the cosmetics industry. In recent years, we have seen a rise in the usage of various nano-substances, including peptides, proteomics, stem cells, and epigenetics. These could directly intervene against the sources of any dermatological phenomenon, be it wrinkles, pigment spots, or acne.

The potential is immense, which is demonstrated by the industrys rising investment and the fact that major cosmetic producers, including LOral, P&G, Dior, and Johnson & Johnson, publish several nanotechnology-related patents every year.

LOral specifically designated a web page to nanoparticles to educate their consumers about the power of these substances in many aspects; from intensifying the shade of mascara to providing a matte finish effect on the skin.

The uses are truly diverse. For example, we can find the adoption of nanoemulsions that encapsulate active ingredients to be carried deeper into hair shafts, or nanosilvers and nanogolds that are known for their antibacterial effects and are used in deodorant or toothpaste.

Nanotechnology is practically used in all everyday products, including moisturizers, haircare, or sunscreens.

In fact, the usage of nanoparticles in sunscreens has perhaps earned the most attention. These SPF creams contain zinc oxide and titanium dioxide as their main compounds. Such products can reflect UV rays, in contrast to the traditional chemical sunscreens that absorb the rays.

Thats why nano-powered sunscreens appear transparent, instead of leaving a white layer on the skin. Yet, this method has been associated with safety concerns, arising from the risk of the particles penetrating tissue and entering the human organism.

While there is still research to be done, an Australian study from last year disproved this notion and asserted that nano-powered sunscreens are unlikely to be harmful.

But its not just healthcare and beauty. Nanotechnology also brings opportunities to conservation and preservation. By being able to disrupt biological processes at the most detailed level, scientists are working to delay wilting and enhance desired processes, such as fostering an environment unfriendly to bacteria.

Specifically, there has been a lot of progress done in the field of food storage and preservation. For example, the encapsulation of nutraceuticals through nanotechnology is a step towards greater food safety and bioavailability, allowing us to benefit from food to its full nutritional potential.

However, similar applications could boost a plethora of other industries, including design and art, education, and science. Laboratories could find easier ways to preserve biological samples, while impressive natural artworks could be exhibited in museums for decades, bringing awe to many generations.

One of the innovations already being put to practice is the NanoFreeze technology, which can directly battle the sources of flower decay. This preservation strategy relies on a uniquely set up freezing process that can halt decomposition.

It kills present microorganisms, stops enzymatic reactions, and establishes a protected environment that prevents the occurrence of parasites. This way, NanoFreeze technology succeeds in maintaining the bloom looking fresh even years after it was cut off, bringing unprecedented possibilities to the floriculture industry and beyond.

From live-saving innovations to more mundane consumer upgrades, nanotechnology presents many opportunities for the future.

While predicted to grow significantly in the upcoming years, its crucial to understand that the innovation still hasnt reached its peak and is yet to experience its full bloom.

Disclosure: This article is brought to you by a client of an Espacio portfolio company

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How nanotechnology is sizing up healthcare, beauty and conservation - The Sociable

ORLANDO, Fla.--(BUSINESS WIRE)--

- Grant is part of the Helping to End Addiction Long-Term Initiative (NIH HEAL Initiative) launched in 2018 -

Hesperos, Inc., (www.hesperosinc.com) announced today the University of Central Florida (UCF) and Hesperos have received the first phase of a $3.8 million milestone-based National Institutes of Health (NIH) grant for research involving the companys human-on-a-chip system. The goal of the research is to better understand how overdosing on opiates works, their impact on multiple organs and the effect of drugs used to treat those overdose episodes including their potential toxicity to organs. James Hickman, Ph.D., Chief Scientist at Hesperos and Professor at UCF, is the principal investigator for the research.

Hesperos, Inc., (www.hesperosinc.com) announced today the University of Central Florida (UCF) received the first phase of a $3.8 million milestone-based National Institutes of Health (NIH) grant for research involving the companys human-on-a-chip system.

"We are grateful to have funding to support research in an area that represents such a large and growing need," said Dr. Hickman. "Our interconnected human-on-a-chip system provides a non-invasive way to emulate the response of compounds among all 'organ' compartments, and to concurrently predict potential toxicity and efficacy of drugs, including opioids and opioid antagonists such as Narcan."

The funding comes from the NIHs Helping to End Addiction Long-term Initiative, or the NIH HEAL Initiative. The initiative aims to improve treatments for chronic pain, curb the rates of opioid use disorder and overdose and achieve long-term recovery from opioid addiction. The National Institutes of Health launched the Helping to End Addiction Long-term Initiative, or NIH HEAL Initiative, in April 2018 to improve prevention and treatment strategies for opioid misuse and addiction and enhance pain management. More information about the grants awarded by the NIH HEAL Initiative can be found here.

Its clear that a multi-pronged scientific approach is needed to reduce the risks of opioids, accelerate development of effective non-opioid therapies for pain and provide more flexible and effective options for treating addiction to opioids, said NIH Director Francis S. Collins, M.D., Ph.D., who launched the initiative in early 2018. This unprecedented investment in the NIH HEAL Initiative demonstrates the commitment to reversing this devastating crisis.

Under this program, Hesperos will build overdose models in a multi-organ system and evaluate the acute and chronic effects of overdose treatments, such as Narcan, on overdose recovery and efficacy. The research will provide insight into the impact of both opiate overdoses and treatment drugs on the kidneys, heart, muscles and liver, as well as explore how these drugs impact the part of the brain that controls breathing to reproduce overdose conditions.

Hesperos seeks to radically change established practice in drug discovery and testing by bypassing animal experiments and extensive clinical trials to provide treatments for diseases and clinical conditions such as overdose. Dr. Hickman developed the human-on-a-chip system at UCF in collaboration with Michael Shuler, President and CEO at Hesperos and Professor Emeritus, Cornell University. UCF licensed the technology to Hesperos, which was co-founded by Dr. Hickman and Dr. Shuler.

Over the past few years we have formed multiple collaborations with companies and nonprofit organizations seeking more efficient and effective alternatives to preclinical evaluation of drugs or toxicity tests on chemicals without lengthy, expensive animal studies, Dr. Shuler said. We recently published results in Nature Scientific Reports and Science Translational Medicine supporting the ability of our system to truly revolutionize the drug discovery process.

About Hesperos:

Hesperos, Inc. is a leader in efforts to characterize an individuals biology with human-on-a-chip microfluidic systems. Founders Michael L. Shuler and James J. Hickman have been at the forefront of every major scientific discovery in this realm, from individual organ-on-a-chip constructs to fully functional, interconnected multi-organ systems. With a mission to revolutionize toxicology testing as well as efficacy evaluation for drug discovery, the company has created pumpless platforms with serum-free cellular mediums that allow multi-organ system communication and integrated computational PKPD modeling of live physiological responses utilizing functional readouts from neurons, cardiac, muscle, barrier tissues and neuromuscular junctions as well as responses from liver, pancreas and barrier tissues. Created from human stem cells, the fully human systems are the first in vitro solutions that accurately utilize in vitro systems to predict in vivo functions without the use of animal models. More information is available at http://www.hesperosinc.com

View source version on businesswire.com: https://www.businesswire.com/news/home/20190926005748/en/

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Hesperos Human-on-a-Chip Technology Awarded First Phase of $3.8 Million Milestone-based NIH Grant to Study Opiate Overdoses in Collaboration With UCF...

CAIRO 22 September 2019: Renowned Professor of Cardiothoracic surgery Magdi Yacoub said that the foundation stone of a heart center in Cairo will be laid soon. The center will provide cardiac care.

In an interview with Egypt Today, the Egyptian-British cardiothoracic surgeonsaid that an inauguration ceremony of the Cairo center will beheldin January 2020, and will beattended by a large number of parliamentarians, senior doctors and statesmen to support the center and urge Egyptians to donate.

The MagdiYacoub Global Heart Foundation launched a campaign in May to raise fund for the new center.

A set of remarkable scientists and public figures took part in the campaign such as Professor MagdyIshak, and Egyptian Ambassador to the United States Yasser Reda, among others.

The MagdiYacoub Global Heart Foundation supports Aswan heart centre in Upper Egypt and is raising funds for the future MagdiYacoubglobal heart centre in Cairo.

Besides providing urgently needed cardiac care, the centers impact the region and continent by advancing scientific understanding through research and building human health capacities with training programs.

The new center will cost an estimate of $150 million and will include 300 beds, hence expected to upgrade network care capacity from 33,000 to 140,000 outpatients and from 4,000 to 17,000 inpatients annually.

Moreover, the training capacity will grow from 550 to over 2300, dramatically increasing the sectors workforce.

Yacoub was among the first three surgeons to perform an open heart surgery in Nigeria in 1974. In 1986, he was part of the team that developed the techniques of the heart-lung transplantation at the National Heart and Lung Institute.

He also led a British research team at Harefield hospital in 2007, aiming to grow a part of the human heart using stem cells. These efforts were all exerted in order to overcome the shortage of heart transplant donations.

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Cairo heart center to be inaugurated January: Magdi Yacoub - Egypttoday

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Global Autologous Stem Cell Based Therapies (Thousands Units) and Revenue (Million USD) Market Split by Product Type such as , Embryonic Stem Cell, Resident Cardiac Stem Cells & Umbilical Cord Blood Stem Cells

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Global Autologous Stem Cell Based Therapies Sales (Thousands Units) by Application (2017-2022)

(2017-2022)

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The report provides a basic overview of the Autologous Stem Cell Based Therapies industry including definitions, classifications, applications and industry chain structure. And development policies and plans are discussed as well as manufacturing processes and capital expenditures.Further it focuses on global major leading industry players with information such as company profiles, product picture and specifications, sales, market share and contact information. Whats more, the Autologous Stem Cell Based Therapies industry development trends and marketing channels are analyzed.The study is organized with the help of primary and secondary data collection including valuable information from key vendors and participants in the industry. It includes historical data from 2012 to 2017 and projected forecasts till 2022 which makes the research study a valuable resource for industry executives, marketing, sales and product managers, consultants, analysts, and other people looking for key industry related data in readily accessible documents with easy to analyze visuals, graphs and tables. The report answers future development trend of Autologous Stem Cell Based Therapies on the basis of stating current situation of the industry in 2017 to assist manufacturers and investment organization to better analyze the development course of Autologous Stem Cell Based Therapies Market.

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Autologous Stem Cell Based Therapies Market Report with Depth Analysis 2019 | Regeneus, Mesoblast - Tech Platform

DURHAM, N.C., Sept. 25, 2019 (GLOBE NEWSWIRE) -- Dance Biopharm, a clinical-stage company reimagining the treatment of chronic diseases with inhaled therapies, today announced it is rebranding as Aerami Therapeutics and moving its headquarters to Durham, North Carolina. The new brand, which will be implemented immediately, comes as the company pursues strategic partners for its lead asset, Dance 501 inhaled human insulin, to progress into pivotal registration studies, and expands its pipeline to include inhaled therapeutics for endocrinology diseases designed to provide patients an alternative to routine injectable treatments.

The new brand better reflects the Companys broadened focus beyond diabetes to include additional chronic conditions that could potentially achieve better treatment efficiency from the patient friendly administration afforded by Aeramis gentle mist, smart inhaler. Aerami plans to advance new pipeline programs in hypoparathyroidism and human growth hormone (HGH) deficiency in addition to its existing inhalable GLP-1 receptor agonist candidate. Each of these programs could enter clinical development in 2020, subject to our ability to obtain additional funding.

We are proud to launch Aerami, which is derived from a combination of Greek and Latin words. Aero means air/wind and ami means friend/friendly. The new brand reflects our commitment to provide patients living with chronic diseases, a patient-friendly alternative to routine injections, with our gentle mist inhaler, said Anne Whitaker, chief executive officer of Aerami Therapeutics. Our initial focus going forward is on rare and severe diseases in the endocrinology, respiratory and cardiovascular space. We have launched new pipeline programs in hypoparathyroidism and human growth hormone deficiency, which could benefit from the frequent pulsatile administration of inhaled parathyroid hormone and HGH, respectively, afforded by our smart inhaler. In addition, we are targeting advancing our inhalable pre-meal GLP-1 receptor agonist into the clinic in 2020, subject to additional funding, for treatment of type 2 diabetes patients who are not meeting their HbA1c goals with their current oral anti-diabetic treatments.

About Aerami Therapeutics

Aerami Therapeutics is a clinical-stage company reimagining the treatment of chronic diseases with inhaled therapies. The Companys gentle mist inhaler is designed with smart technology to optimize the precise delivery of biologic therapies through the lungs. In addition to its Phase 3-ready inhaled insulin product Dance-501, Aerami is building a diverse pipeline of inhaled therapies with an initial focus on rare and severe diseases in the endocrinology, respiratory and cardiovascular space, where its platform and expertise present a unique opportunity to achieve better treatment efficiency and patient experience. Aerami has initiated development programs for inhaled glucagon-like peptide-1 (GLP-1) for the treatment of type 2 diabetes, inhaled parathyroid hormone (PTH) for the treatment of hypoparathyroidism and inhaled human growth hormone (HGH) for the treatment of HGH deficiency.

For more information, visit: https://www.aerami.com/.

Investor Contact:Jeremy FefferLifeSci Advisors, LLC(212) 915-2568jeremy@lifesciadvisors.com

Media Contact:Gloria GasaaturaLifeSci Public Relations(646) 627-8387ggasaatura@lifescipublicrelations.com

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Dance Biopharm Announces Rebranding to Aerami Therapeutics Reflecting a Broadened Strategic Focus on Inhaled Therapies for Chronic Diseases - Yahoo...

Fountain Hills Internal Medicine and Pediatrics has expanded to include two naturopathic physicians and the services of a cardiologist/vascular specialist.

Joining medical director Dr. Kaveh Karandish are licensed naturopathic physicians Dr. Tess Price and Dr. Chad Patterson.

Dr. Moshen Sharifi, director of Arizona Cardiovascular Consultants & Vein Clinic, also is a staff member.

An open house is scheduled from 6 to 9 p.m. on Thursday, Oct. 10, to meet the new health care providers. Light refreshments will be served.

Since Karandish acquired ownership of the practice last January, he has been assessing the communitys medical needs. He said his office combines preventative and integrative medicine.

He created a six-day-a-week urgent walk-in clinic and established home visits within a five-mile radius of town for patients who are physically unable to travel to his office at 13620 N. Saguaro Blvd., suite 100.

Cosmetic procedures, including services and skin products, are provided. Laser therapy hair removal is a service.

Bio-Identical hormone pellets designed to optimize hormones that a body naturally produces are a new procedure for men and women. Karandish said they have the ability to improve the quality of life for some patients.

A weight loss program to meet individual needs is provided. Under the category of regenerative medicine, the practice can customize platelet-rich plasma and stem cell treatments. Acupuncture is another new service.

Dr. Patterson specializes in pediatric patients between birth and 18 years of age. His primary method of treatment includes nutritional supplements and herbal remedies.

He was born in Denmark and grew up in Carlisle, Penn. A Scottsdale resident, he completed his undergraduate degree majoring in biology from Pennsylvania State University. Post-baccalaureate education took place at Lake Erie College of Osteopathic Medicine. He earned his doctorate in naturopathic medicine from Southwest College of Naturopathic Medicine in Tempe.

Dr. Prices bachelor degree in veterinary science prepared her to enter the work force. She taught in the public and private education sector and counseled college students before returning to school. She also received her doctoral degree in medicine at Southwest College of Naturopathic Medicine.

She has been involved in domestic violence shelters as well as an in-patient treatment facility for addictions, pain and mental health.

She also specializes in gastrointestinal disorders, thyroid dysfunction, mental health issues and preventative medicine.

Dr. Sharifi is an adjunct professor at A.T. Still University in Mesa and is board certified in interventional cardiology, cardiovascular diseases, vascular medicine, internal medicine and nuclear cardiology.

The practices telephone number is 480-837-6800.

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Medical practice adds providers, services | Business - FH Times

Intrauterine devices, more commonly known as IUDs, are the most effective and reversible forms of birth control, according to the Centers For Disease Control and Prevention. If you've decided to use an IUD as your birth control method, you're probably wondering if the insertion process is painful, how long you can have it in, and how soon you can get pregnant if you decide to have it removed. To answer the latter, POPSUGAR enlisted Leah Millheiser, MD, ob-gyn, FACOG, and director of the female sexual medicine program at Stanford Health Care.

As a refresher, "An IUD is a tiny device that's placed in your uterus to prevent pregnancy," Planned Parenthood explains. It's a small piece of plastic that's shaped like a "T," and there are five different FDA-approved brands available in the US: Paragard, Mirena, Kyleena, Liletta, and Skyla.

Brand aside, there are two types of IUDs you can choose from: copper, such as the Paragard, or hormonal, such as the Mirena, Kyleena, Liletta, and Skyla options. The Paragard IUD is wrapped in copper, which acts as a spermicide, according to the US Department of Health and Human Services (HHS), and can prevent pregnancy for up to 12 years.

According to Planned Parenthood, the hormonal IUDs release the hormone progestin to prevent pregnancy. "The progestin causes cervical mucus to thicken and the lining of the uterus to thin," according to the HHS. Because of this, the sperm is unable to reach and fertilize the egg. The Mirena and Liletta IUDs work for up to seven years, Kyleena works for up to five years, and Skyla works for up to three years.

Planned Parenthood said, "IUDs are one of the best birth control methods out there more than 99 percent effective," but if you're considering having a child or switching your birth control method, here's what you need to know about how quickly you can get pregnant.

"You can get pregnant right away," Dr. Millheiser said. Whether you have a hormonal IUD or a copper IUD, neither prevents ovulation, she explained. "Essentially, you can get it taken out and get pregnant very quickly," she added. If you have a hormonal IUD inserted, your fertility should return within a month, according to Dr. Millheiser. "We [ob-gyns] tell people it's, for the most part, an immediate return to fertility. So, when you're due to ovulate, you're going to ovulate."

If you're wondering why you can get pregnant so quickly, it's because the primary method of the IUD is to prevent the sperm and the egg from meeting and fertilizing in the fallopian tube, Dr. Millheiser explained. If the egg does get fertilized, the IUD prevents it from sticking to the uterine wall, she said.

Even though the IUD is the most effective form of birth control, aside from abstinence, it's important you check your IUD, making sure you can feel the two strings that hang down into your vaginal canal to prevent pregnancy. There's a possibility you may not be able to feel your strings, and in that case, you should use a backup form of birth control and/or contact your medical provider to make sure your IUD hasn't fallen out of your uterus or perforated your cervix or uterus.

If you're interested in learning more about the birth control methods available to you, consult your medical provider or a local sexual healthcare clinic.

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You Can Get Pregnant "Right Away" Once Your IUD Is Removed Here's What You Should Know - POPSUGAR

My niece was diagnosed with Type 1 diabetes when she was 12. The previously robustly healthy girl was suddenly hungry all the time, extremely thirsty, losing weight, feeling fatigued, experiencing vision changes and generally not feeling well.

One day it all came to a head and her parents rushed her to the emergency room. After a very long and scary day of tests, the reason behind all those symptoms became clear. She was diagnosed with Type 1 diabetes.

Type 1 diabetes is an autoimmune disorder (meaning your immune system attacks the bodys own cells) that results from the bodys inability to produce insulin, a hormone that turns food into energy. It affects 1.3 million people in the U.S. and is on the rise, according to the Barbara Davis Center for Diabetes at the University of Colorado Anschutz Medical Campus, one of the largest diabetes research facilities in the world.

If you have Type 1, your pancreas isnt making enough or any insulin. The exact cause of Type 1 diabetes is unknown. Risk factors for Type 1 in children include family history, genetic susceptibility and race (its more common in white children), according to the Mayo Clinic. However, most (90%) of children with diabetes do not have a family member with the disease and often go undiagnosed until they are very sick, according to the BDC. Type 1 can strike children and adults out of the blue, and, while manageable through insulin therapy, can be difficult to manage.

Type 2 diabetes is the kind thats preventable through lifestyle changes such as weight loss. If you have Type 1 you are insulin-dependent for life. There is no cure.

My niece, who is now 17, was a once a happy-go-lucky girl who was afraid of getting a shot at the doctor. Now she literally carries a burden with her wherever she goes, using an insulin pump that supplies her with the right amount of insulin. Some people have to self-inject insulin the old-school way. The pump is a newer technology that some people with Type 1 use in tandem with constant blood-glucose monitoring and carbohydrate counting (to determine how much insulin is needed).

Its a daily science experiment in which the patient is the guinea pig. Blood sugar has to be frequently monitored to make sure the pump is operating correctly. The site where its attached to the body (typically a fatty spot such as hip or upper arm) can get scar tissue buildup, and must be changed regularly. The wearer must pre-bolus or manually pump insulin to balance out a high-glycemic meal. Theres no taking a day off its a constant vigil without which the pump user can go high or low on blood sugar. Either way is bad for the body, and can be dangerous.

These are tough lessons for anyone to manage on a daily basis, let alone a child. When my niece was diagnosed, her parents were happy to have an answer for all the health issues shed been having. But now they had to learn quickly how to manage her disease.

Fortunately, the Barbara Davis Center provides instruction. The center cares for 3,600 children and 2,400 adults with diabetes in the Rocky Mountain region, per its website. Its financially supported by the Childrens Diabetes Foundation, established by Barbara and Marvin Davis in 1977. Important research is also conducted at the center.

That research there are currently 81 studies being done at Barbara Davis Center is paramount to finding a cure for Type 1 diabetes. The center also supports free Autoimmunity Screening for Kids for undiagnosed celiac disease and Type 1 diabetes in Colorado children ages 1-17. For more information and to find an ASK screening location, visit ASKhealth.org or call the hotline, 303-724-1275.

Last Sunday I joined about 10 other family members and friends (and thousands of others) on my nieces team at the JDRF One Walk in Denver. The Juvenile Diabetes Research Foundation raised more than $550,000 from the Denver walk alone. There are 200 JDRF walks nationwide this year, including one at Rock Ledge Ranch in Colorado Springs on Oct. 12. The foundation has raised more than $2 billion to date to fund research to create a world without Type 1.

JDRF funds research that transforms the lives of people with T1D. We want a cure, and we wont stop until we find one but we need your help. Along the way, we will continue to drive scientific progress that delivers new treatments and therapies to help people with T1D stay healthy and live longer, states jdrf.org.

Some of JDRFs goals are to create a future that holds treatment options such as the artificial pancreas and beta-cell replacement. And, ultimately, prevention of Type 1 diabetes, so kids like my niece can stop worrying about their health and get back to being kids.

Michelle Karas has called the Pikes Peak region home for more than four years. She became editor of Pikes Peak Newspapers in June. Contact Michelle with letters to the editor, guest columns or story ideas at michelle.karas@pikespeaknewspapers.com.

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From the Editor: Research is imperative to finding a cure for Type 1 diabetes - Colorado Springs Gazette

Mark Mahoney(Photo: Mark Mahoney)

As we move toward the beginning of fall, the issue of understanding the importance of reducing belly fat through a healthier lifestyle can lead to an improved quality of life. Why is it important to address excess belly (or visceral) fat? And, what are some simple tips to consider for addressing this health problem? Taking proactive steps now can prove beneficial to your future longevity.

Visceral fat is stored in a person's abdominal cavity and is also known as 'active fat' as it influences how hormones function in the body. An excess of visceral fat can, therefore, have potentially dangerous consequences. Because visceral fat is in the abdominal cavity, it is close to many vital organs, such as the pancreas, liver, and intestines.

The higher the amount of visceral fat a person stores, the more at risk they are for certain health complications which may include many-varied negative health conditions such as heart attacks, heart disease, type 2 diabetes, heightened blood pressure, stroke, breast and colorectal cancer and Alzheimers disease.

Get more fiber:You dont have to consume a bag of prunes. Leafy greens, whole grains, nuts, and beans are all good for keeping away the fat that stays deep in your belly. Thats called visceral fat, and its the most dangerous kind because it can wrap around major organs, including your liver, pancreas, and kidneys.

Forget these two things:There are no super foods that burn off visceral fat. And you cant tone it away with specific moves like crunches. Instead, look for ways to improve your eating habits and add activity every day. Think about your average week. Where might you be able to make some changes?

Limiting saturated fats:While anyone can have too much visceral fat, its more likely if youve got a lot of weight to lose. As you start to take those pounds off, it will help your whole body, including belly fat thats hidden out of your sight.

Limit the saturated kind thats in animal foods, coconut and palm oils, and full-fat dairy. Keep the portions of those foods smaller than you might normally do, for instance. And check nutrition labels to see how many calories and how much fat is in a serving. Look for fats that are better for you, too, like those from plant foods or fish such as salmon, tuna, and mackerel that are rich in omega-3s.

Sleep: An important formula:When it comes to weight gain, shut-eye is a bit like porridge: Too little less than 5 hours may mean more belly fat. But too much more than 8 hours can do that, too. Just right seems to be around 6-8 hours. If you dont sleep that much now, or if you tend to toss and turn, try to go to bed a little earlier, relax before bedtime, keep your bedroom cool, and try not to text and email right before you turn in.

Forget the quick fix:Sorry, but cosmetic surgery isnt the solution here. Liposuction doesnt reach inside the abdominal wall. So it cant get rid of visceral belly fat. Likewise, crash diets arent the solution, either. Youre too likely to go off them. The slower, steadier option lifestyle changes that you can commit to for a long time really is the best bet.

Keep calm:Are you stressed out? That can make you eat more fat and sugar, and unleash the stress hormone cortisol, which can boost belly fat. Stress also can make you sleep less, exercise less, and drink more alcohol which can add belly fat, too. Its a great reason to take up meditation, work out, listen to music you love, or find other healthy ways to unwind and relax.

Rethink your drink:Whether its a latte, a regular soda, a mug of beer, or a glass of wine, its got calories. And when youre trying to unwind the numbers on the scale, water (or a smaller glass of your favorite beverage) might be a better choice. If you drink alcohol, remember that it just might make you throw your willpower out the window when you order your meal, too.

Dont smoke:As if you need another reason to quit. Smoking makes you more likely to store fat in your belly, rather than your hips and thighs. And thats bad. Oh, and its also a cause of diabetes, cancer, heart disease, and lung disease. And you get the idea. If youve tried before, try again. Tell your doctor, so you can get pointers on what might help you quit for good.

Weight gain around the middle can have negative health affects. Recommendations include adopting a whole-foods diet cutting out processed, refined, stripped-of-fiber carbs and advocating healthy fats and lean proteins.(Photo: Martinina, Getty Images/iStockphoto)

Importance of waist size:A good method is to measure your waist. If youre a woman, you want that number to be 35 inches or less. Men can go up to 40 inches. You may lower your chance of having a heart attack, a stroke, or possibly certain types of cancer. A tape measure cant check on visceral fat. But along with the scale, it can help you track your weight loss.

Dont try to outrun it:Research shows that a few quick bursts of high-intensity exercise such as a 30-second sprint or intense pull-up sets may be more effective, and easier to fit into your schedule than long runs. You can add bursts of higher intensity to any workout. Just speed up or work harder for a brief time, then drop back to a mellower pace, and repeat.

Weights:Think about hitting the gym instead of the trail. In one study, healthy middle-aged men who did 20 minutes of daily weight training gained less abdominal fat than men who spent the same time doing aerobic exercises, such as biking. Strength training is also good for women and it wont make you bulky. You still need to do some cardio, but make sure strength training is in the mix.

Visceral fat is fat that we cannot see, so it is not always easy to know whether a person has an excess of it. Because the associated health risks can be severe, it is essential for those who suspect their visceral fat levels are high to seek advice from a health professional.

Usually, it is possible to avoid high levels of visceral fat by leading a healthy and active lifestyle. Those who do store dangerous amounts of visceral fat can reduce their levels by making positive changes to their lifestyle. Three important changes include eating a nutritious, low-fat diet, undertaking appropriate types of exercise, and lowering stress levels.

Additional in-depth information on addressing visceral fat can be found at the following sites:

https://www.health.harvard.edu/staying-healthy/abdominal-fat-and-what-to-do-about-it

https://www.mayoclinic.org/healthy-lifestyle/mens-health/in-depth/belly-fat/art-20045685

Thanks to Medical News Today and the Mayo Clinic and Harvard Health for much of the content here.

Consult a qualified health professional before undertaking any specific type of high intensity exercise and if you have any questions related to increasing your physical activity level.

Mark A. Mahoney, Ph.D. has been a Registered Dietitian/Nutritionist (RDN) for over 35 years and completed graduate studies in Nutrition & Public Health at Columbia University. He can be reached at marqos69@hotmail.com.

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Take slow, steady approach to beat stubborn belly fat - Tallahassee.com

Unexplained weight loss: 5 reasons youre losing weight without trying; when to see a doctor  |  Photo Credit: Getty Images

New Delhi: Many people who want to get fitter and leaner find it hard to shed those extra pounds. On the other hand, there are some people who have unexplained weight loss, which can be a cause for concern. While the benefits of maintaining a healthy weight go far beyond improved health and appearance, losing weight without trying may signal an underlying condition. Health experts warned that unintentional weight loss can be a symptom of a serious illness, including heart failure, diabetes or cancer.

Anybody can experience unexplained weight loss especially after a life-changing or stressful event. But, in older people, losing weight of even less than 5 per cent of the body weight or 10 pounds may indicate that something isnt just right, as per Cleveland Clinic. Below are some reasons why unexplained weight loss could be a serious problem.

Congestive heart failure

Weight loss can be a sign of congestive heart failure (CHF), a condition in which the heart cant pump enough blood and oxygen to the bodys tissues. Heart failure is associated with weight loss as the inflammation in the damaged heart tissue can also increase the bodys metabolism. Other symptoms of CHFinclude:

Diabetes

Often people with type 1 diabetes, prior to diagnosis, have unexplained weight loss - although it can also affect people with type 2 diabetes. According to the Diabetes.co.uk, insufficient insulin stops the body from getting glucose from the blood and into the cells of the body to use as energy. Hence, the body begins to burn fat and muscle for energy, which leads to a reduction in overall body weight. Other symptoms of type 1 diabetes are:

Cancer

Unexplained weight loss of 10 pounds or more could be one of the first signs of cancer, as per the American Cancer Society. In fact, 40% of people experienced weight loss when first diagnosed with cancer, a group of diseases that cause abnormal cells to divide and spread uncontrollably, destroying body tissue. Unusual weight loss is common especially in cancers of the pancreas, lung, stomach, and oesophagus. Other early symptoms of cancer include -

Hyperthyroidism

Hyperthyroidism, or overactive thyroid, is a condition in which your thyroid gland produces too much of thyroxine, a hormone secreted by the thyroid gland into the bloodstream. It can accelerate your bodys metabolism, causing your body to burn calories quickly despite having a good appetite. This can result in unintentional weight loss. Other symptoms of an overactive thyroid include:

Chronic obstructive pulmonary disease

COPD is an umbrella term used to describe chronic inflammatory lung diseases such as emphysema and chronic bronchitis, causing obstructed airflow from the lungs. And that effort to breathe burns a lot of calories - way more than someone with healthy lungs, Albert Rizzo, MD, chief of pulmonary and critical care medicine at Christiana Care Health System, told WebMD. The Cleveland Clinic said damaged lungs can burn as much as 10 times more calories than normal, healthy lungs, which can lead to unintentional weight loss.

Other symptoms of COPD include:

Apart from this, there are many other health conditions that can cause unusual weight loss such as - tuberculosis, inflammatory bowel disease, rheumatoid arthritis, depression, etc.

The fact is its quite normal for anybody to experience weight fluctuations. However, if youre losing weight without changing your habits or trying through diet/exercise, it may signal something more serious. It is advisable to seek medical help if a person is experiencing a 5% weight loss of their body weight in 6-12 months.

Disclaimer: Tips and suggestions mentioned in the article are for general information purposes only and should not be construed as professional medical advice. Always consult your doctor or a professional healthcare provider if you have any specific questions about any medical matter.

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Losing weight not always good, could be a sign of ailment- when to see a doctor - Times Now

HTF MI recently introduced new title on Global Active Pharmaceutical Ingredients (API) Market Study 2016-2026, by Segment (Vitamin, Antibiotics), by Market (Hospital, Clinic), by Company (Teva Active Pharmaceutical Industries Limited, DSM,) from its database. The report provides study with in-depth overview, describing about the Product / Industry Scope and elaborates market outlook and status to 2025. The Report gives you competition analysis of top manufacturer with sales volume, price, revenue (Million USD) and market share, the top players including Teva Active Pharmaceutical Industries Limited, DSM, Novartis, Roche, Johnson & Johnson, Dr. Reddy s, Pfizer, Bayer, BASF, Cambrex, Sun Pharmaceutical Industries, Lonza group, Boehringer Ingelheim GmbH, Aurobindo pharma

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Active Pharmaceutical Ingredients (API) Market Next Big Thing | Major Giants DSM, Novartis, Roche, Johnson & Johnson - Industry News Stock

We often complain about our body fat, especially if it is hard to lose. In our complaining, we fail to realize that body fat is needed for the human body to regulate several bodily functions. Fat cells can be found in the arms, thighs, buttocks, and in the belly surrounding the organs. These fat cells are located in these areas of the body storing energy to be used at a later time. More than that our body fat is needed to absorb the fat-soluble vitamins A, D, E, and K. Also, what most people dont know is that having a healthy amount of body fat is required for blood sugar regulation. Body fat is also needed for the regulation of human growth hormone. Having a healthy amount of body is important for the human body. Then again, for every benefit of body fat, there is a health risk. According to the American Council on Exercise, having a body fat percentage higher than the recommended range could put a person at risk for over half a dozen health conditions. That is why it is important to lose excess body fat reducing the chances of developing a health condition. However, for some this can be easier said than done as body fat is not always easy to lose. By eating smart, balancing your hormones, and making behavioral changes it can be possible to lose body fat when previous methods have failed.

When losing weight is presenting as a challenge there are 5 scientifically proven ways that can help by reducing body fat. By employing these practices long-term it is possible to lose the desired amount of body fat and keep it off.

It is not easy to be appreciative of what our body fat does for us when there are several reasons why to much body fat can be harmful to our health. It can be even harder on those trying to lose weight are having difficulty in losing body fat. Losing body fat is not always easy, but can become possible by using any one of the 5 weight loss techniques listed above. All 5 of these techniques focus on managing ones hormones, controlling behavior, and changing the way a person thinks to improve the chances of weight loss. They have all been scientifically proven to increase the chances of losing body fat in order to lose weight, even if previous attempts at weight loss have failed.

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5 Scientifically Proven Ways to Improve Weight Loss Efforts When All Else Failed - Boston Sports Extra

September 25 2019, 12:01am,The Times

Carol Midgley

Aside from that other big story (something about Boris Johnson?) youll have noticed that primary pupils are being given self-touching lessons. They are being taught that stimulating their private parts is not dirty, but really very normal, although not in class when everyone is watching.

Madness. I mean, what a waste of time. Because as any parent of a teenage boy will confirm, they work all that stuff out pretty damn well by themselves. Give it a few years, head teachers, and they may not be acing maths, but theyll certainly be achieving an A* in onanism with all the happy socks under the bed to prove it. So relax. Save a few quid on your budgets. Because its balls.

But then so much of

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My husband had better be a sapiosexual like Mark Ronson - The Times

The neurobiologist Belinda Pletzer from the University of Salzburg is researching the psychological effect of the contraceptive pill. She is focusing on the neurobiological effects on the structures inside the brain and whether these are reversible. She is particularly interested in puberty as one of the most sensitive phases of brain development.

There are about 9000 known studies on health risks and side effects of the pill. In addition to physical symptoms such as weight gain, high blood pressure and thrombosis, the psychological effects have also been studied. Until now, however, only about fifteen studies have explored the influence that the contraceptive pill has on the brain. As she has studied amongst other things biology and psychology, observing the causes of psychological effects on the brain is also an appropriate method for Pletzer.

In her study, the researcher is looking at contraceptives which contain two different active ingredients:

Women react very differently emotionally when taking the various birth control pills available. Pletzer adds:

For some women, using these may cause depressive moods. For others, they have a stabilizing effect. After all, some birth control pills are prescribed for premenstrual syndrome (PMS) as well.

PMS refers to complex physical and emotional discomfort associated with a womans menstrual cycle.

The project aims to reveal what distinguishes women who can tolerate the contraceptive pill easily, from women who cannot. It also aims to study how the various contraceptives affect the brain during sensitive periods of the brains development, such as puberty.

The tests are carried out using fMRI scans at the Christian Doppler Clinic. The test subjects have to solve various cognitive problems and their scans must be recorded in order to determine whether taking the pill alters the brain structure or brain activity.

The tests are performed before, during and after taking contraceptives. After a contraceptive pill has been discontinued, a check is carried out to see whether the effect is reversible.

Pletzer works at the Centre for Cognitive Neuroscience in Salzburg. Her project was awarded a 1.5 million ERC Starting Grant from the European Research Council. The exceptional 36-year-old researcher studied biology, psychology, philosophy and mathematics and has two doctorates. She is also the mother of four children. The funding runs for five years and will allow her to conduct a comprehensive study with 300 test subjects.

An interview with Belinda Pletzer:

When we talk about the psyche, we think about emotional well-being. We have known since the 1960s that the pill has an effect on the psyche. There are also studies on this, but the results are contradictory. Some studies have found an increase in depression, whereas others have shown stabilizing effects on emotional well-being. Both have merit, women have different reactions. These effects have been observed by gynecologists and proven in studies.

I am concerned with the neurobiological structures in the brain and as of yet there are practically no relevant studies on this. For example, there are studies that examined in group comparisons whether the brain structure of women who take the pill differs from that of those who do not. This is questionable from a methodological point of view. Every person is different.

We are conducting a longitudinal study and comparing the development of womens brain structure before, during and after use of the pill.

Because there are scarcely any studies in this field, we are trying to cover cognition in as comprehensibly as possible. As a general rule, these are the aspects spatial, verbal and memory. We test:

For facial recognition, we did a preliminary study wherein we were able to show that facial recognition skills are improved when taking certain types of contraceptive pills. Face recognition has a correlation with the gray mass in the area of the brain responsible for face recognition.

Separately from us, another group looked at brain activity, which is also correlated. The longer women take the pill, the greater its effect.

Face recognition should be included in studies that examined the influence of the contraceptive pill on memory. In fact, and with a considerable amount of circumspection, it could be said that the common denominator among the few available studies is that taking the contraceptive pill seems to slightly improve memory function.

This does not mean that taking the contraceptive pill is either good or bad, but merely that it may have an effect. As every woman reacts differently to using the pill and there are still very few relevant studies, it is not yet possible to make recommendations.

Our hypotheses are based on findings concerning the effect of endogenous hormones on the brain. We have looked at a number of brain regions that have consistently responded to hormones in a similar way across a variety of studies and in different test subjects. When estradiol levels rise, there is more gray matter and more activity in the hippocampus. As the pill usually contains a very strong synthetic estrogen (ethylene estradiol), we can expect a very similar effect. But this still has yet to be proven.

Note: estradiol is a sex hormone and the most effective natural estrogen (estrogen) in comparison with estrone and estriol. It is produced mainly in the ovarian follicles.

Also of interest:

How the brain distinguishes between voice and sound

Study using AI: mens and womens brains are different

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The influence of contraceptives on cognitive ability - Innovation Origins

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20 Small Things That Supercharge Weight Loss - CafeMom

Biomedical engineers at Duke University have used a previously unexplored CRISPR technology to accurately regulate and edit genomes in human cells.

With this new approach, the researchers hope to dramatically expand the CRISPR-based tools available to biomedical engineers, opening up a new and diverse frontier of genome engineering technologies.

In a study appearing on Sept. 23 in Nature Biotechnology, Charles Gersbach, the Rooney Family Associate Professor of Biomedical Engineering at Duke, and Adrian Oliver, a post-doctoral fellow in the Gersbach lab who led the project, describe how they successfully harnessed Class 1 CRISPR systems to turn target genes on and off and edit the epigenome in human cells for the first time.

CRISPR-Cas is a defense system in which bacteria use RNA molecules and CRISPR-associated (Cas) proteins to target and destroy the DNA of invading viruses. The discovery of this phenomenon and the repurposing of the molecular machinery set off a genome-editing revolution as researchers learned how to wield the tool to specifically target and edit DNA in human cells.

CRISPR-Cas9, the most commonly used genome editing tool today, is categorized as a Class 2 CRISPR system. Class 2 systems are less common in the bacterial world, but they are theoretically simpler to work with, as they rely on only one Cas protein to target and cleave DNA.

Class 1 systems are not so simple, relying on multiple proteins working together in a complex called Cascade (CRISPR-associated complex for antiviral defense) to target DNA. After binding, Cascade recruits a Cas3 protein that cuts the DNA.

"If you were to look at the individual CRISPR systems of all the bacteria in the world, nearly 90 percent are Class 1 systems," said Gersbach. "CRISPR-Cas biology is an incredible source for biotechnology tools, but until recently everyone has only been looking at a small slice of the pie."

To demonstrate the capabilities of the Class 1 system, Oliver attached gene activators to specific sites along a type I E. coli Cascade complex and targeted the system to bind gene promoters, which regulate gene expression levels. Because she did not include the Cas3 protein in the experiment, there was no cutting of the DNA and no change to underlying DNA sequence. The experiment showed that the Cascade activator not only binds to the correct site and can turn up the levels of the target gene, but does so with accuracy and specificity comparable to CRISPR/Cas9.

Oliver repeated the process using type I Cascade complexes from an additional bacterial strain that was particularly robust in working at a variety of target sites. She also showed that the activator domain could be swapped for a repressor to turn target genes off. Again, the researchers noted accuracy and specificity comparable to CRISPR/Cas9 methods.

"We have found Cascade's structure to be remarkably modular, allowing for a variety of sites to attach activators or repressors, which are great tools for altering gene expression in human cells," Oliver said. "The flexible nature of Cascade makes it a promising genome engineering technology."

Gersbach and Oliver were encouraged to investigate the more complicated Class 1 CRISPR systems by their collaborators at nearby North Carolina State University, Professors Rodolphe Barrangou and Chase Beisel, who is now at the Helmholtz Centre for Infection Research in Germany. Barrangou is a microbiologist who has studied the natural biology of diverse CRISPR defense mechanisms for nearly two decades, and Beisel is a chemical engineer who has worked with Barrangou on engineering microorganisms with Class 1 CRISPR systems. They were both curious whether Gersbach's lab could use these systems in human cells similar to their work with Cas9.

"This work and the resulting technologies are a fantastic example of how collaboration across disciplines and across universities in the North Carolina Research Triangle can be highly innovative and productive" says Barrangou, the Todd R. Klaenhammer Distinguished Professor in Probiotics Research at North Carolina State University.

Now, the team is optimistic that their study, and the related work of others in the field, will incentivize new research into Class 1 CRISPR systems.

"The purpose of this project was to explore the diversity of CRISPR systems," said Gersbach. "There have been thousands of papers about CRISPR-Cas9 in the last decade, and yet we're constantly learning new things about it. With this study we're applying that mindset to the other 90% of what's out there."

So far, the team has shown that these Class 1 systems are comparable to to CRISPR-Cas9 in terms of accuracy and application. As they consider future directions, they are curious to explore how these systems differ from their Class 2 counterparts, and how these differences could prove useful for biotechnology applications.

The team is also interested in studying how Class 1 systems could address general challenges for CRISPR-Cas research, especially issues that complicate potential therapeutic applications, like immune responses to Cas proteins and concurrently using multiple types of CRISPR for different genome engineering functions.

"We know CRISPR could have a big impact on human health," said Gersbach. "But we're still at the very beginning of understanding how CRISPR is going to be used, what it can do, and what systems are available to us. We expect that this new tool will enable new areas of genome engineering."

Reference: Pickar-Oliver. 2019.Targeted transcriptional modulation with type I CRISPRCas systems in human cells. Nature Biotechnology. DOI: https://doi.org/10.1038/s41587-019-0235-7.

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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CRISPR Tool Opens Up a New Frontier of Genome Engineering Technologies - Technology Networks

The U.S. Patent and Trademark Office (USPTO) today granted the University of California (UC) and its partners, the University of Vienna and Emmanuelle Charpentier, a new CRISPR-Cas9 patent, bringing the teams continually expanding patent portfolio to 15.

Jennifer Doudna, co-inventor of the CRISPR-Cas9 gene-editing tool, in 2014, with a model of the complex on her computer screen. (UC Berkeley photo courtesy of Cailey Cotner)

U.S. Patent 10,421,980 covers compositions of certain DNA-targeting RNAs that contain RNA duplexes of defined lengths that hybridize with Cas9 and target a desired DNA sequence. The patent also covers methods of targeting and binding a target DNA, modifying a target DNA, or modulating transcription from a target DNA wherein the method comprises contacting a target DNA with a complex that includes a Cas9 protein and a DNA-targeting RNA.

In the coming months, based on applications allowed by the USPTO, UCs CRISPR-Cas9 patent portfolio will increase to 18. Together, these patents cover compositions and methods for CRISPR-Cas9 gene-editing, including targeting and editing genes and modulating transcription in any setting, such as within plant, animal and human cells.

With every patent that issues, UC strengthens its position as the leader in CRISPR-Cas9 intellectual property in the United States, said Eldora Ellison, the lead patent strategist on CRISPR-Cas9 matters for UC and a director at Sterne, Kessler, Goldstein & Fox. We are steadfast in our commitment to developing a comprehensive patent portfolio that protects the groundbreaking work of the Doudna-Charpentier team on CRISPR-Cas9.

The team that invented the CRISPR-Cas9 DNA-targeting technology included Doudna and Martin Jinek at UC Berkeley; Charpentier, then at Umea University in Sweden and now director of the Max Planck Institute for Infection Biology in Germany; and Krzysztof Chylinski of the University of Vienna. The methods covered by todays patent, as well as the other methods claimed in UCs previously issued patents and those set to issue, were included among the CRISPR-Cas9 gene editing technology work disclosed first by the Doudna-Charpentier team in its May 25, 2012, priority patent application.

Doudna is a UC Berkeley professor of molecular and cell biology and of chemistry, a Howard Hughes Medical Institute investigator and holder of the Li Ka Shing Chancellors Chair in Biomedical and Health Sciences. She also is executive director of the Innovative Genomics Institute, a faculty scientist at Lawrence Berkeley National Laboratory and a senior investigator at the Gladstone Institutes in San Francisco.

Additional CRISPR-Cas9 patents in this teams portfolio include 10,000,772; 10,113,167; 10,227,611; 10,266,850; 10,301,651; 10,308,961; 10,337,029; 10,351,878; 10,358,658; 10,358,659; 10,385,360; 10,400,253; 10,407,697; and 10,415,061. These patents are not a part of the PTABs recently declared interference between 14 UC patent applications and multiple previously issued Broad Institute patents and one application, which jeopardizes essentially all of the Broads CRISPR patents involving eukaryotic cells.

International patent offices have also recognized the pioneering innovations of the Doudna-Charpentier team, in addition to the 15 patents granted in the U.S. so far. The European Patent Office (representing more than 30 countries), as well as patent offices in the United Kingdom, China, Japan, Australia, New Zealand, Mexico, and other countries, have issued patents for the use of CRISPR-Cas9 gene editing in all types of cells.

University of California has a long-standing commitment to develop and apply its patented technologies, including CRISPR-Cas9, for the betterment of humankind. Consistent with its open-licensing policies, UC allows nonprofit institutions, including academic institutions, to use the technology for non-commercial educational and research purposes.

In the case of CRISPR-Cas9, UC has also encouraged widespread commercialization of the technology through its exclusive license with Caribou Biosciences, Inc. of Berkeley, California. Caribou has sublicensed this patent family to numerous companies worldwide, including Intellia Therapeutics, Inc. for certain human therapeutic applications. Additionally, Dr. Charpentier has licensed the technology to CRISPR Therapeutics AG and ERS Genomics Limited.

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University of California awarded 15th U.S. CRISPR-Cas9 patent - UC Berkeley

On one level, Malakkar Vohryzek always knew what was wrong with him. For as long as he can remember hes now 43 the sun has been his enemy, making angry-looking moles pop up on his white-as-a-fish-belly skin like toxic mushrooms after a downpour.

At age 9, he bit one off. Since his teens he has had moles removed as regularly as other kids got haircuts, hoping to catch the growths before they became malignant. Because of his skins extreme sensitivity to sunlight, he takes every UV-blocking precaution, from SPF 60 sunscreen and hats and other cover-ups to, as a 19-year-old, working the graveyard shift as a waiter at Dennys so he could commute in darkness.

But there is no name for what Vohryzek has, and no cure. There is no known inherited genetic mutation that might explain why just a few ultraviolet rays make his skin cells proliferate wildly, forming moles. One of these days, Vohryzek is convinced, hell overlook one, or wait too long before seeing a dermatologist, and hell wake up with malignant melanoma. That cancer, if it metastasizes, is usually fatal.

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Vohryzek, a legal researcher and IT consultant in southern California, has therefore taken matters into his own hands. For the last few months, he has blitzed scientists, biotechnology companies, and biohackers as far away as Sendai, Japan, with email pleas to please CRISPR him. I just want to live, Vohryzek told STAT in an interview.

It was perhaps inevitable that the campaign to give patients the right to try experimental drugs before FDA approval enshrined in a 2018 law would combine with unbounded optimism about the potential curative power of genome-editing technology to send someone like Vohryzek on this quest. In California, lawmakers were so concerned about people biohacking themselves or others with unproven therapies that they passed a law this summer banning it.

Id expect to see more people like Vohryzek, said bioethicist Alison Bateman-House of New York University, an expert on avenues for patients to access experimental therapies. Although parents are increasingly asking scientists for access to experimental compounds that have never even been tested in animals, in order to save their children from a devastating disease, there are likely to be even more such pleas for genetic technologies.

Its so intuitively simple: replace or correct a gene thats not functioning properly, Bateman-House said. There is so much hype, more and more people will think, I want that.

In his early 30s, Vohryzek became increasingly frantic about UV-induced DNA damage, which he fears is worsening with age. Hed spent much of his 20s in federal prison for distributing LSD, after which he legally changed his first name to a bastardization of the Arabic (malak al qur) and Hebrew for Angel of Truth and took his mothers last name. After his release, he found that moles were erupting with frightening frequency, especially on his arms.

In 2017, he read that biohacker Josiah Zayner, who sells genetic engineering kits and lessons through his company The Odin, injected himself with a purportedly muscle-boosting CRISPR cocktail onstage at a biotechnology conference. That inspired me, Vohryzek said though Zayners stunt didnt work and he began combing genetics papers for research on radiation protection.

Earlier this year, he happened on a gene that he believes will save him, one from the tiny, rotund, eight-legged water bear aka moss piglet, aka tardigrade which protects it from the damaging effects of radiation. A study from Japan reported that scientists had sequenced the genome of Ramazzottius varieornatus, a species of the famously resilient tardigrade, and identified a previously unknown gene. It turned out to code for a protein they called Dsup (for damage suppressor).

What caught Vohryzeks eye was what happened when Takuma Hashimoto of the University of Tokyo and his colleagues slipped the tardigrade gene into human cells growing in lab dishes and then bombarded them with X-rays. Photon for photon, X-rays are hundreds of times more powerful than the suns ultraviolet rays. Human cells genetically engineered to express tardigrade Dsup withstood 40% more radiation than regular human cells.

Dsup works by minimizing the harm to genes, apparently, by encasing cells DNA, much like a lead shield in a nuclear reactor. As a result, radiation doesnt break the strands of the double helix a breach that can trigger cancer. Dsup, Vohryzek thought, could protect him from solar UV and therefore melanoma.

For the last few months, he has been asking scientists and companies if theyll give him the biological supplies he would need he isnt always clear on what those might be to receive the tardigrade gene, using CRISPR or some other technology to slip it into his cells.

Hashimotos experiment, Vohryzek told STAT, demonstrates that Im not proposing something insane. I want to participate in [the] use of CRISPR on full genome gene insertion.

In July, he emailed his request to Hashimoto, explaining that he will die soon from skin cancer unless he receives the Dsup gene. If you know a team that can [use] CRISPR to insert the Dsup production into my genome, Vohryzek promised, he would sign an agreement not to hold them responsible for any mishaps. If the experiment killed him, he said, he would donate his body to science so researchers could figure out what happened.

In fact, genome editing technologies such as CRISPR only tweak what already exists in a genome. It can alter a DNA letter, or nucleotide, to transform the gene from a disease-causing form to a healthy one. It can snip out regions from the former, disabling them and leaving only the healthy version (people inherit two copies of every gene, one from mom and one from dad). It can insert a few nucleotides in place of a misspelled, disease-causing segment.

But it cannot insert a completely novel gene. Thats called genetic engineering. Although there are now two approved gene therapies in the U.S., for a form of blindness and for spinal muscular atrophy, this intervention is considered less precise and more prone to problems than genome editing.

None of that has dampened Vohryzeks interest. Although his chief motivation is avoiding melanoma I just know that eventually the roll of the genetic dice will come up snake eyes, and I will die, he said he also believes that becoming a human guinea pig would advance science.

CRISPR science has the potential to save billions of lives, and end misery for billions more, he said. I have hundreds of reasons to willingly contribute my own body for furthering its research, and no reason at all not to.

He has received almost no replies to his requests, and the ones hes gotten have hardly been encouraging. While technically feasible there are many ethical and legal implications to attempting this, wrote an executive at the genetics supply company Atum. Im not sure what sort of help we can give you with this project. To be honest, it seems more like a science fiction project than a commercially viable product. We deal mostly with the latter.

It seems unlikely that any academic or biotech scientists will grant Vohryzeks wish. On the other hand, the birth of CRISPR babiesalso seemed unlikely, until scientist He Jiankui produced two of them in China last year. Just as that bombshell sent tremors through legitimate developers of CRISPR therapies, so could a rogue researcher putting a water bear gene into Vohryzek, said NYUs Bateman-House: Im very worried about systemic ramifications, including shutting down gene therapy everywhere.

None of the biohacker collectives contacted by STAT said it had been asked by patients for help with do-it-yourself genome editing, but Bateman-House suspects that it is just a matter of time. Earlier this month, Vohryzek asked a friend who was attending a biohackers gathering in Las Vegas to see whether any of them might be willing to give him the Dsup gene, though he said he would prefer the professionals to a garage DIYer. In November, he plans to attend a meeting of a DIY collective in Seattle to see if my experimental treatment is feasible for them.

If I die of melanoma, it wont help anyone, he said. If I die because of an experimental treatment, it will at least help science.

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California man pleads with scientists to 'CRISPR me' - STAT - STAT

In the volatile world of biotech, investors can find a number of promising markets that could see significant growth in the years to come. Gene editing is one of these markets that has tremendous potential for growth, with some research reports showing the sector could reach $9.66 billion by 2025. Considering that in 2018, the gene-editing market was worth only $3.7 billion; this works out to an impressive 14.7% annual growth rate.

While few biotech stocks are developing gene-editing therapies, the complexity of the subject matter can make it difficult for investors to categorize where an individual company stands in relation to its competition.CRISPR Therapeutics (NASDAQ:CRSP)has a head start. Having developed the CRISPR Cas9 gene-editing technology, CRISPR has quickly made a name for itself in the biotech world. Since then, CRISPR Therapeutics has earned a market cap of $2.65 billion despite only having a handful of early clinical trials to show at the moment.

Amid this wave of excitement, shares of CRISPR have risen by 61.8% so far in 2019. While CRISPR will have to clear a few hurdles before its gene-editing drugs can hit the markets, there's a strong case to be made for this stock becoming a leader in the space. Let's take a look at some of the main therapies CRISPR is developing at the moment and how exactly it compares to its competition.

IMAGE SOURCE: GETTY IMAGES.

CRISPR's main strategy with its gene-editing technology has been to target major genetically based disorders. Its flagship drug, an anti-sickle cell medication known as CTX001, is undergoing a phase 1 clinical trial that, if successful, would be highly lucrative for the company. Approximately 100,000 Americans are diagnosed with sickle cell disease currently, with children being especially vulnerable to the condition.

At the moment, there's no cure for the disease, and researchers are currently exploring the possibility of bone marrow transplants as a therapeutic option. A gene-editing drug that could treat sickle cell anemia would be a home run for CRISPR if it were approved for human use.

Earlier this year, CRISPR announced the first sickle-cell patient had been treated with CTX001 in its clinical trial, and the research team is waiting to see if there are any long-term effects of the treatment. Although CRISPR has said that the clinical trial could be concluded in 2022 and a potential rollout could quickly follow, in the world of clinical trials, that's still plenty of time for things to change.

CRISPR also has a number of early-stage clinical trials underway for its cancer immunotherapies. CTX110 is a drug that is being tested as a potential treatment against cancerous tumors, while CTX120 and CTX130 are other candidate drugs for various cancer types. Cancer immunology is an already-massive market that's estimated to grow to $126.9 billion by 2026, representing a 9.6% compound annual growth rate. However, there's already a fair bit of competition in this space.

Specifically, CTX110 is what's referred to as an allogeneic chimeric antigen receptor T-cell (CAR-T) therapy. Healthcare giantsGilead (NASDAQ:GILD) and Novartis (NYSE:NVS) have their own CAR-T therapies, but the problem they face is that this treatment is extremely expensive for patients, not to mention being quite slow.

CAR-T therapies require scientists to remove immune cells from a patient, teach those cells to better fight cancer cells, and then reintroduce them into the patient's system. Understandably, this is both time-consuming and expensive, with treatments costing hundreds of thousands of dollars per patient.

One solution to these problems is to develop CAR-T drugs from donor cells. This process, referred to as allogeneic, could lead to complications from patients whose immune systems react against or even reject the donor cells. This is where CRISPR comes in, as its gene-editing technology can significantly minimize the chance of rejection.

Ananalyst at Needham named Alan Carr said,"An off-the-shelf allogeneic product presents several advantages and we believe CRISPR is uniquely positioned to succeed in the space, given rights are wholly owned and the simplicity and flexibility of CRISPR technology to make multiple simultaneous edits."

While the market for gene-editing technology is excellent, the last question worth asking is where CRISPR Therapeutics stands in comparison to its rivals. CRISPR's potential competitors include Intellia Therapeutics (NASDAQ:NTLA) and Editas Medicine (NASDAQ:EDIT), both companies working in the gene-editing field. The latter is developing its own sickle cell treatment to rival CTX001. However, Editas is also working on a gene-editing drug called EDIT-101 that would help babies with protein deficiencies in their retinas leading to blindness. So while there is some overlap between the two companies, there are also areas in which the two aren't in direct competition.

CRISPR also enjoys a cushier financial position than the other two companies, with $427.9 million in cash reserves compared to Editas' $317.9 million and Intellia's $275.8 million. However, CRISPR Therapeutics doesn't have as strong an intellectual-property portfolio as some of its competitors; Editas owns more than 70 issued patents and 600 pending patent applications, not to mention the rights to technology owned by institutions such as MIT and Harvard.

Overall, it's hard to say which gene-editing company will end up reigning supreme in the years to come. I would take a shotgun approach, investing a little in most of the promising gene-editing biotech stocks in the market right now while acknowledging that many will likely fail. CRISPR Therapeutics definitely makes the list of gene-editing stocks worth buying, although only for investors willing to jump into a high-risk, high-reward type of investment.

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Is CRISPR Therapeutics a Buy? - Motley Fool