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New gene therapy proves promising as hemophilia treatment
Date:12/11/2013

CHAPEL HILL, N.C. Researchers at the UNC School of Medicine and the Medical College of Wisconsin found that a new kind of gene therapy led to a dramatic decline in bleeding events in dogs with naturally occurring hemophilia A, a serious and costly bleeding condition that affects about 50,000 people in the United States and millions more around the world.

Before the gene treatment, the animals experienced about five serious bleeding events a year. After receiving the novel gene therapy, though, they experienced substantially fewer bleeding events over three years, as reported in the journal Nature Communications.

"The promise and the hope for gene therapy is that people with hemophilia would be given a single therapeutic injection and then would express the protein they are missing for an extended period of time, ideally for years or even their entire lifetimes," said Tim Nichols, director of the Francis Owen Blood Research Laboratory at UNC and co-author of the paper. The hope is that after successful gene therapy, people with hemophilia would experience far fewer bleeding events because their blood would clot better.

People with hemophilia A lack the coagulation factor VIII in their blood plasma the liquid in which red, white, and platelet cells are suspended.

"Bleeding events in hemophilia are severe, and without prompt factor VIII replacement, the disease can be crippling or fatal," said Nichols, a professor of medicine and pathology. "The random and spontaneous nature of the bleeding is a major challenge for people with hemophilia and their families."

In underdeveloped countries, people with hemophilia and many undiagnosed people typically die from bleeding in their late teens or early 20s. In developed countries, patients usually live fairly normal lives, as long as they receive preventive injections of recombinant protein therapy a few times a week. The disease requires life-long management that
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Contact: Mark Derewicz
mark.derewicz@unch.unc.edu
919-923-0959
University of North Carolina Health Care
Source:Eurekalert  

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New gene therapy proves promising as hemophilia treatment
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