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Hemophilia is a rare, inherited bleeding disorder in which your blood doesn’t clot normally. If you have hemophilia, you may bleed for a longer time than others after an injury. You also may bleed internally, especially in your knees, ankles, and elbows. This bleeding can damage your organs or tissues and, sometimes, be fatal.
Hemophilia B patients produce near-normal levels of clotting factor IX after gene therapy, study shows

Hemophilia B patients produce near-normal levels of clotting factor IX after gene therapy, study shows

Researchers are reporting the highest and most sustained levels to date of the essential blood-clotting factor IX in patients with the inherited bleeding disorder hemophilia B. [More]
Penn researchers use CRISPR/Cas9 gene targeting approach to treat hemophilia B in mice

Penn researchers use CRISPR/Cas9 gene targeting approach to treat hemophilia B in mice

CRISPR/Cas9, a powerful genome editing tool, is showing promise for efficient correction of disease-causing mutations. [More]
UT Austin researchers develop new oral capsule for treating hemophilia patients

UT Austin researchers develop new oral capsule for treating hemophilia patients

In the near future, hemophiliacs could be able to treat their disease by simply swallowing a capsule. [More]
ASGCT seeks to educate public and policy-makers on fundamentals of gene editing

ASGCT seeks to educate public and policy-makers on fundamentals of gene editing

On Nov. 21, 2016, the American Society of Gene & Cell Therapy released Therapeutic Gene Editing: an ASGCT White Paper, intended to provide policy-makers, patient advocates, and the interested public with the necessary background information in anticipation of an upcoming consensus report on human gene editing from the National Academy of Sciences and National Academy of Medicine (NAS), expected to be released in early 2017. [More]
TSRI scientists unravel mystery of ‘food coma’ phenomenon

TSRI scientists unravel mystery of ‘food coma’ phenomenon

Anyone who has drifted into a fuzzy-headed stupor after a large holiday meal is familiar with the condition commonly known as a "food coma." [More]
TSRI scientists offer novel structure-based drug design strategy for better breast cancer treatment

TSRI scientists offer novel structure-based drug design strategy for better breast cancer treatment

While there have been advances in the treatment of hormone-driven breast cancer, resistance to these therapies remains a significant problem. [More]
TSRI study reveals how circadian clocks may influence cancer growth

TSRI study reveals how circadian clocks may influence cancer growth

A new study led by scientists at The Scripps Research Institute describes an unexpected role for proteins involved with our daily "circadian" clocks in influencing cancer growth. [More]
TSRI study unravels how protein is directly responsible for sensing touch

TSRI study unravels how protein is directly responsible for sensing touch

A new study led by scientists at The Scripps Research Institute reveals that a protein first discovered at TSRI in 2010 is directly responsible for sensing touch. [More]
New European study emphasizes need to enhance standard of haemophilia care in real life

New European study emphasizes need to enhance standard of haemophilia care in real life

Swedish Orphan Biovitrum AB (publ) announces the results from a new European study that assessed the efficacy of haemophilia care in real life. [More]
TSRI study suggests new approach to limit tumor growth by targeting immune system cells

TSRI study suggests new approach to limit tumor growth by targeting immune system cells

A new study led by scientists at The Scripps Research Institute suggests there may be a way to limit tumor growth by targeting immune system cells called macrophages. [More]
TSRI scientists receive NIH grants for computational biology research

TSRI scientists receive NIH grants for computational biology research

Three groups at The Scripps Research Institute have been awarded grants from the National Institutes of Health to develop methods for computational modeling and to apply them to cutting-edge systems in biology and health. [More]
TSRI scientists develop useful technique to unmask new functional features of human proteins

TSRI scientists develop useful technique to unmask new functional features of human proteins

Scientists at The Scripps Research Institute have developed a broadly useful method to unmask new functional features of human proteins. [More]
Two leading non-profit organizations sign strategic affiliation to hasten drug development

Two leading non-profit organizations sign strategic affiliation to hasten drug development

The Scripps Research Institute and the California Institute for Biomedical Research - two leading non-profit research organizations - today announced the signing of a strategic affiliation that combines the two organizations into a new biomedical research entity with the tools and know-how to rapidly translate its scientific discoveries into life-saving medicines for the public benefit. [More]
TSRI scientists elucidate how Zika virus attacks the brains of newborns

TSRI scientists elucidate how Zika virus attacks the brains of newborns

In a study that could one day help eliminate the tragic birth defects caused by Zika virus, scientists from the Florida campus of The Scripps Research Institute have elucidated how the virus attacks the brains of newborns, information that could accelerate the development of treatments. [More]
New virtual reality games create better patient-experience for kids during infusions

New virtual reality games create better patient-experience for kids during infusions

As a nurse clinician in the comprehensive hemophilia treatment center at Nationwide Children's Hospital for nearly 30 years, Charmaine Biega, RN, has watched her patients endure hundreds of needle sticks for infusions and other procedures which can mean tears, frustration, wiggling and - in some cases - lifelong anxiety about the medical system and treatments that patients with hemophilia need to survive. [More]
TSRI researchers find potential new weapon to combat C. difficile infections

TSRI researchers find potential new weapon to combat C. difficile infections

Scientists at The Scripps Research Institute have discovered a potential new weapon against Clostridium difficile, a bacterium that causes hundreds of thousands of severe intestinal infections in the U.S. every year and is frequently fatal. [More]
TSRI study sheds light on how mitochondrial calcium can affect learning and memory

TSRI study sheds light on how mitochondrial calcium can affect learning and memory

While calcium's importance for our bones and teeth is well known, its role in neurons—in particular, its effects on processes such as learning and memory—has been less well defined. [More]
TSRI scientists find evidence supporting new therapeutic strategy against cocaine addiction

TSRI scientists find evidence supporting new therapeutic strategy against cocaine addiction

An international team led by scientists at The Scripps Research Institute has found strong evidence supporting a new strategy against drug addiction. [More]
New study suggests increased levels of hypocretin in the brain may play role in cocaine addiction

New study suggests increased levels of hypocretin in the brain may play role in cocaine addiction

A new study from scientists at The Scripps Research Institute, funded by the National Institutes of Health's National Institute on Drug Abuse and National Institute on Alcohol Abuse and Alcoholism, suggests that increased levels of a molecule in the brain, called hypocretin, may contribute to cocaine addiction. [More]
Scientists link malfunctioning molecular pathways to specific heart abnormalities in SCA

Scientists link malfunctioning molecular pathways to specific heart abnormalities in SCA

Patients with sickle cell anemia (SCA) develop heart complications and nearly a quarter die a sudden death. Now, researchers have linked malfunctioning molecular pathways to specific heart anomalies in SCA that result from progressive fibrosis and result in sudden death. [More]
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