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Research highlights need to address hemophilia in developing world
When modern medicine finds a way to treat a medical condition, people often think that the problem is solved. But we also have to find ways to get that treatment into the hands of those who need it.   view more (2009-11-16)

Jumping genes discovery 'challenges current assumptions'
Jumping genes do most of their jumping, not during the development of sperm and egg cells, but during the development of the embryo itself.   view more (2009-06-12)

Gene therapy technique thwarts cancer by cutting off tumor blood supply
University of Florida researchers have come up with a new gene therapy method to disrupt cancer growth by using a synthetic protein to induce blood clotting that cuts off a tumor's blood and nutrient supply.   view more (2009-06-12)

Gene therapy demonstrates benefit in patients with rheumatoid arthritis
Researchers have reported the first clinical evidence that gene therapy reduces symptoms in patients with rheumatoid arthritis, an important milestone for this promising treatment which has endured a sometimes turbulent past.   view more (2009-01-27)

Redesigned protein accelerates blood clotting
Researchers have made several, subtle changes in the structure of a key protein, dramatically increasing its ability to drive blood clotting, according to a study published in a December edition of the Journal of Thrombosis and Haemostasis.   view more (2008-12-23)

Mapping a clan of mobile selfish genes
Much of human DNA is the genetic equivalent of e-mail spam: short repeated sequences that have no obvious function other than making more of themselves.   view more (2008-10-23)

Blood-clotting protein modified for people with hard-to-treat hemophilia
Pathologists at The University of Texas Health Science Center at Houston have developed a chemically modified protein that may help people with a hard-to-treat form of a genetic bleeding disorder known as Hemophilia A.   view more (2008-05-20)

UF researchers develop improved gene therapy agent
Replacing one amino acid on the surface of a virus that shepherds corrective genes into cells could be the breakthrough scientists have needed to make gene therapy a more viable option for treating genetic diseases such as hemophilia, University of Florida researchers say.   view more (2008-05-20)

Skin flaps deliver cancer-fighting therapy, ASPS study reveals
Using gene therapy, plastic surgeons have delivered cancer fighting proteins through skin flaps placed on cancerous tumors on rats with a 79 percent reduction in tumor volume.   view more (2008-05-09)

NHLBI Issues First U.S. von Willebrand Disease Clinical Practice Guidelines
The National Heart, Lung, and Blood Institute (NHLBI) of the National Institutes of Health, today issued the first clinical guidelines in the United States for the diagnosis and management of von Willebrand Disease (VWD), the most common inherited bleeding disorder.   view more (2008-03-03)

First demonstration of muscle restoration in an animal model of Duchenne muscular dystrophy
Using a new type of drug that targets a specific genetic defect, researchers at the University of Pennsylvania School of Medicine, along with colleagues at PTC Therapeutics Inc. and the University of Massachusetts Medical School, have for the first time demonstrated restoration of muscle function in a mouse model of Duchenne's muscular dystrophy... view more... (2007-04-23)

Fruit fly gene research may shed light on human disease processes
Those small fruit flies buzzing around your bananas are more than pests—they may be allies in a fruitful search for clues to human diseases caused when genes malfunction.   view more (2007-03-28)

A transplant in time
In hemophilia, a mutated gene prevents the production of a critical blood-clotting protein. Treatments for hemophilia and other such genetic diseases, when they exist, may consist of risky blood transfusions or expensive enzyme replacement therapy.   view more (2007-01-02)

Jumping gene could provide non-viral alternative for gene therapy
A jumping gene first identified in a cabbage-eating moth may one day provide a safer, target-specific alternative to viruses for gene therapy.   view more (2006-09-26)

Rodent's bizarre traits deepen mystery of genetics, evolution
A shadowy rodent has potential to shed light on human genetics and the mysteries of evolution.   view more (2006-09-18)

Emory scientists develop new map of genetic variation in human genome
Emory University scientists have identified and created a map of more than 400,000 insertions and deletions (INDELs) in the human genome that signal a little-explored type of genetic difference among individuals.   view more (2006-08-11)

Novel gene therapy may lead to cure in hemophilia A patients
A discovery by Medical College of Wisconsin and BloodCenter of Wisconsin researchers in Milwaukee may be a key to a permanent genetic cure for hemophilia A patients, including a subset who do not respond to conventional blood transfusions.   view more (2006-07-05)

Baxter and Jerini advance program to develop non-intravenous haemophilia therapy
Baxter AG and Jerini AG today announced progress in their collaborative research program to develop a non-intravenous therapy for the treatment of hemophilia. Extensive in vitro and in vivo analysis has identified several synthetic lead molecules with promising pharmacological properties.   view more (2006-06-20)

For Stanford scientists, RNAi gene therapy takes two steps forward, one step back
Three years ago Mark Kay, MD, PhD, published the first results showing that a hot new biological phenomenon called RNA interference was an effective gene-therapy technique in mice.   view more (2006-05-25)

Ancient DNA helps UF researchers unearth potential hemophilia therapy
A cut can be life-threatening for people with hemophilia, whose bodies don't produce enough of a protein that prevents prolonged bleeding.   view more (2006-02-28)
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