Hemophilia News and Research

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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.

TSRI scientists develop new tool to uncover molecular details of protein structure

Scientists on the Florida campus of The Scripps Research Institute have developed a new tool for studying the molecular details of protein structure.

5 Jan 2017

New TSRI study sheds light on how the brain stores memories

A new study led by scientists at The Scripps Research Institute (TSRI) sheds light on how the brain stores memories.

4 Jan 2017

AAV-based gene therapy to treat liver disorders advances into human testing

Liver-directed gene therapy delivered using adeno-associated viral (AAV) vectors to treat diseases such as hemophilia have advanced into human testing.

30 Dec 2016

TSRI scientists develop new approach for understanding diverse effects of endocrine-disruptors

Breast cancer researchers from the Florida campus of The Scripps Research Institute (TSRI) have developed a novel approach for identifying how chemicals in the environment--called environmental estrogens--can produce infertility, abnormal reproductive development, including "precocious puberty," and promote breast cancer.

30 Dec 2016

A new strategy to fight prostate cancer

A new study led by scientists from the Florida campus of The Scripps Research Institute (TSRI) sheds light on a signaling circuit in cells that drives therapy resistance in prostate cancer.

30 Dec 2016

Researchers working to develop pill for inherited bleeding disorders

Motivated by the tribulations of hemophilia patients and their families, researchers funded by the National Institute of Biomedical Imaging and Bioengineering are working to develop a pill to treat this serious inherited bleeding disorder.

23 Dec 2016

TSRI scientists shed light on how antiviral drug stops influenza infections

A new study led by scientists at The Scripps Research Institute (TSRI) is the first to show exactly how the drug Arbidol stops influenza infections. The research reveals that Arbidol stops the virus from entering host cells by binding within a recessed pocket on the virus.

22 Dec 2016

TSRI scientist receives Outstanding Investigator Grant to focus on memory formation research

Ron Davis, chair of the Department of Neuroscience on the Florida campus of The Scripps Research Institute has been awarded a $5 million Outstanding Investigator Grant, one of the first of its kind, by the National Institute of Neurological Disorders and Stroke of the National Institutes of Health.

20 Dec 2016

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.

4 Dec 2016

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.

1 Dec 2016

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.

29 Nov 2016

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.

24 Nov 2016

Hemophilia News and Research

Further Reading

TSRI scientists develop new tool to uncover molecular details of protein structure

New TSRI study sheds light on how the brain stores memories

AAV-based gene therapy to treat liver disorders advances into human testing

TSRI scientists develop new approach for understanding diverse effects of endocrine-disruptors

A new strategy to fight prostate cancer

Researchers working to develop pill for inherited bleeding disorders

TSRI scientists shed light on how antiviral drug stops influenza infections

TSRI scientist receives Outstanding Investigator Grant to focus on memory formation research

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

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

UT Austin researchers develop new oral capsule for treating hemophilia patients

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

TSRI scientists unravel mystery of ‘food coma’ phenomenon

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

TSRI study reveals how circadian clocks may influence cancer growth

TSRI study unravels how protein is directly responsible for sensing touch

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

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

TSRI scientists receive NIH grants for computational biology research

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

Designing fluidic architecture for scalable diagnostic platforms

Evaluating new approaches for rare disease genomics

Expanding access to NMR with the Fourier 80 Duo

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