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 researchers reveal how viruses incapacitate bacterial defense system

Picture bacteria and viruses locked in an arms race. For many bacteria, one line of defense against viral infection is a sophisticated RNA-guided "immune system" called CRISPR-Cas.

28 Mar 2017

Scientists identify molecules that may be key to drug resistance in estrogen-driven breast cancers

Three-quarters of all breast cancer tumors are driven by the hormone estrogen. These tumors are frequently treated with drugs to suppress estrogen receptor activity, but unfortunately, at least half of patients do not respond to these treatments, leaving them with drug-resistant tumors and few options.

20 Mar 2017

TSRI chemists devise versatile molecule-building tool for creating new drugs and chemical products

Chemists at The Scripps Research Institute have devised a versatile molecule-building tool for creating new drugs and other chemical products.

9 Mar 2017

TSRI scientists discover how key protein establishes proper vision

Scientists from the Florida campus of The Scripps Research Institute have discovered how a protein called α2δ4 establishes proper vision.

6 Mar 2017

UMass Amherst study highlights unmet drug needs of patients with rare diseases

A new analysis by health policy researchers at the University of Massachusetts Amherst who studied the effectiveness of the 1983 Orphan Drug Act finds that current incentives "are not sufficiently stimulating orphan drug development" by pharmaceutical companies, and patients with rare diseases and conditions still have unmet needs. Further, barriers to ethical and timely access remain.

16 Feb 2017

Salk scientists use mRNA to successfully treat hemophilia B in mice

FedEx, UPS, DHL--when it comes to sending packages, choices abound. But the most important delivery service you may not have heard of mRNA.

16 Feb 2017

TSRI scientists describe how key molecules in the brain work together to trigger intense anxiety

In a new study, researchers at The Scripps Research Institute have described how two important molecules in the brain work together to trigger intense anxiety.

13 Feb 2017

Plant-based drug for hemophilia shows promise in animal models

People with hemophilia require regular infusions of clotting factor to prevent them from experiencing uncontrolled bleeding. But a significant fraction develop antibodies against the clotting factor, essentially experiencing an allergic reaction to the very treatment that can prolong their lives.

13 Feb 2017

TSRI chemists create new technique for constructing chiral drug molecules

Chemists at The Scripps Research Institute (TSRI) have invented a new technique for constructing chiral drug molecules.

3 Feb 2017

Deep brain stimulation can reduce heroin relapse in rat models of addiction, TSRI study shows

Scientists at The Scripps Research Institute have found that deep brain stimulation (DBS) can greatly reduce the compulsion to use heroin in standard rat models of addiction.

2 Feb 2017

TSRI study offers first look at early stages of brain development in patients with Fragile X syndrome

A new study led by scientists at The Scripps Research Institute is giving researchers a first look at the early stages of brain development in patients with Fragile X syndrome, a disorder that causes mild to severe intellectual disability and is the most common genetic cause of autism spectrum disorder.

31 Jan 2017

New gene therapy for Pompe disease may replace currently available treatments

After decades investigating a rare, life-threatening condition that cripples the muscles, Duke Health researchers have developed a gene therapy they hope could enhance or even replace the only FDA-approved treatment currently available to patients.

27 Jan 2017

TSRI biologists uncover new mechanism behind sense of touch

Biologists on the Florida campus of The Scripps Research Institute have discovered a new mechanism that likely underlies how we feel force or touch. Their study suggests that "rafts" of fatty lipids on the cell surface act as compartments to keep certain enzymes from mixing with their binding partners.

6 Jan 2017

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