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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 identify GlyRS protein that launches cancer growth

TSRI scientists identify GlyRS protein that launches cancer growth

Scientists at The Scripps Research Institute have identified a protein that launches cancer growth and appears to contribute to higher mortality in breast cancer patients. [More]
Study reveals surprising diversity in single neuronal transcriptomes of the brain

Study reveals surprising diversity in single neuronal transcriptomes of the brain

A team of scientists at The Scripps Research Institute, University of California, San Diego and Illumina, Inc., has completed the first large-scale assessment of single neuronal "transcriptomes." [More]
TSRI scientists develop novel technique for finding drug candidates

TSRI scientists develop novel technique for finding drug candidates

Scientists at The Scripps Research Institute have developed a powerful new method for finding drug candidates that bind to specific proteins. [More]
Study provides more insight into treatment options for severe hemophilia A

Study provides more insight into treatment options for severe hemophilia A

Families of children with severe hemophilia A may want to take a fresh look at treatment options from human plasma. A study published in the New England Journal of Medicine on May 26 showed that participants who received a recombinant therapy— the present standard in the United States — developed antibodies or "inhibitors" to the treatments at almost twice the rate as those whose treatments were made from human plasma. [More]
Scientists use new technique to repair fibrotic liver cells within the organ

Scientists use new technique to repair fibrotic liver cells within the organ

Advances in stem cell research have made it possible to convert patients' skin cells into heart cells, kidney cells, liver cells and more in the lab dish, giving researchers hope that one day such cells could replace organ transplantation for patients with organ failure. [More]
SIPPET study may have implications for treatment of patients with severe hemophilia A

SIPPET study may have implications for treatment of patients with severe hemophilia A

SIPPET, a study which involved 42 centers in 14 countries in Europe, North and South America, Africa and Asia was designed to definitively settle the long-debated question whether factor VIII concentrates from different sources (plasma-derived containing VWF or recombinant technology) differ in risk of inhibitor development in previously untreated children (PUPs) with severe hemophilia A. [More]
Common pain and anti-inflammation drugs may slow cancer growth

Common pain and anti-inflammation drugs may slow cancer growth

Scientists from the Florida campus of The Scripps Research Institute have found that one of the most widely prescribed pain and anti-inflammation drugs slows the growth rate of a specific kind of cancer in animal models and suggests the medication could have the same effect on other types of tumors. [More]
Jumping genes may play crucial role in generation of cancer

Jumping genes may play crucial role in generation of cancer

For more than 50 years, scientists have known of the existence of "jumping genes," strands of DNA material that can move from one location in the genome to another. [More]
TSRI scientists design potential drug candidate to treat triple negative breast cancer

TSRI scientists design potential drug candidate to treat triple negative breast cancer

In a development that could lead to a new generation of drugs to precisely treat a range of diseases, scientists from the Florida campus of The Scripps Research Institute have for the first time designed a drug candidate that decreases the growth of tumor cells in animal models in one of the hardest to treat cancers—triple negative breast cancer. [More]
TSRI study reveals important traits in LCMV, Lassa virus

TSRI study reveals important traits in LCMV, Lassa virus

For the first time, scientists at The Scripps Research Institute (TSRI) have solved the structure of the biological machinery used by a common virus to recognize and attack human host cells. [More]
Wellderly study finds link between cognitive decline genes and healthy aging

Wellderly study finds link between cognitive decline genes and healthy aging

An eight-year-long accrual and analysis of the whole genome sequences of healthy elderly people, or "Wellderly," has revealed a higher-than-normal presence of genetic variants offering protection from cognitive decline, researchers from the Scripps Translational Science Institute (STSI) reported today in the journal Cell. [More]
European Medicines Agency approves first-ever treatment for hereditary factor X deficiency

European Medicines Agency approves first-ever treatment for hereditary factor X deficiency

Bio Products Laboratory, Limited (BPL) today announced that the European Medicines Agency has granted marketing authorisation for Coagadex. [More]
TSRI study shows hollowed-out version of CPMV could be effective in human therapies

TSRI study shows hollowed-out version of CPMV could be effective in human therapies

Viruses aren't always bad. In fact, scientists can harness the capabilities of some viruses for good—modifying the viruses to carry drug molecules, for example. [More]
Scientists identify two enzymes that appear to play role in metabolism, inflammation

Scientists identify two enzymes that appear to play role in metabolism, inflammation

A team led by scientists at The Scripps Research Institute and the Salk Institute for Biological Studies has discovered two enzymes that appear to play a role in metabolism and inflammation—and might someday be targeted with drugs to treat type 2 diabetes and inflammatory disorders. [More]
Engineered HIV vaccine protein may prevent HIV infection

Engineered HIV vaccine protein may prevent HIV infection

Some people infected with HIV naturally produce antibodies that effectively neutralize many strains of the rapidly mutating virus, and scientists are working to develop a vaccine capable of inducing such "broadly neutralizing" antibodies that can prevent HIV infection. [More]
TSRI scientists develop new process to synthesize naturally occurring compound phorbol

TSRI scientists develop new process to synthesize naturally occurring compound phorbol

In a landmark feat of chemical synthesis, scientists at The Scripps Research Institute have developed a 19-step process for making the naturally occurring compound phorbol in the laboratory, in quantities that are useful for pharmaceutical research. [More]
Research: X and Y DNA swapping may occur more often than previously thought

Research: X and Y DNA swapping may occur more often than previously thought

It turns out that the rigid "line in the sand" over which the human sex chromosomes---the Y and X--- go to avoid crossing over is a bit blurrier than previously thought. Contrary to the current scientific consensus, Arizona State University assistant professor Melissa Wilson Sayres has led a research team that has shown that X and Y DNA swapping may occur much more often. [More]
UC San Diego Health participates in nationwide clinical study on hemophilia B gene therapy

UC San Diego Health participates in nationwide clinical study on hemophilia B gene therapy

The Hemophilia and Thrombosis Treatment Center at UC San Diego Health has joined a nationwide clinical trial testing a potential gene therapy that may one day provide a better and long-lasting treatment for people with hemophilia B. [More]
Novel compound shows promise as potential treatment for acute myeloid leukemia

Novel compound shows promise as potential treatment for acute myeloid leukemia

A novel compound has shown promise in preclinical studies as a treatment for acute myeloid leukemia, more than doubling median days of survival even in a drug-resistant form of the disease. [More]
Inherited gene after black plague may help treat HIV patients co-infected with hepatitis C

Inherited gene after black plague may help treat HIV patients co-infected with hepatitis C

The Black Death swept Europe in the 14th century eliminating up to half of the population but it left genetic clues that now may aid a University of Cincinnati researcher in treating HIV patients co-infected with hepatitis C using an anti-retroviral drug therapy. [More]
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