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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)

Gene therapy for muscular dystrophy fixes frail muscle cells in animal model, Stanford study finds
A new gene therapy technique that has shown promise in skin disease and hemophilia might one day be useful for treating muscular dystrophy, according to a new study by researchers at Stanford University School of Medicine.   view more (2005-12-27)

Gene therapy advance treats hemophilia in mouse models
A virus that typically infects insects could help with the development of gene therapy treatment for Hemophilia A, a condition in which even a bump on the knee can cause serious internal bleeding in people.   view more (2005-08-23)

LSU scientists develop new theory about human genome evolution
A group of LSU researchers, led by biological sciences Professor Mark Batzer, have unraveled the details of a 25-million-year-old evolutionary process in the human genome. Their study focused on the origin and spread of transposable elements in the genome, many of which are known to be related to certain genetic disorders, such as hemophilia.   view more (2005-07-01)

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)

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)

HIV inserts into human genome using a DNA-associated protein
A human DNA-associated protein called LEDGF is the first such molecule found to control the location of HIV integration in human cells.   view more (2005-11-28)

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)

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)

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)

Scientists find genetic pathway that could lead to drugs for kidney disease
Scientists at the University of California, Santa Barbara have reported a discovery at the cellular level that suggests possibilities for drug therapy for kidney disease.   view more (2006-02-01)

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)

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)

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)

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)

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)

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)

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)

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)
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