Researchers identify promising gene therapy for DMD

Researchers identify promising gene therapy for DMD
23. June 2010 02:51


CureDuchenne, the Orange County non-profit focused on finding a cure for Duchenne muscular dystrophy (DMD) is one step closer to that goal through the work of Dr. Jeff Chamberlain.

“The focus of the trial will be on how the patient's immune system will respond to the gene delivery”

Dr. Chamberlain, of the University of Washington, in partnership with Dr. Stephen Tapscott, of the Fred Hutchinson Cancer Research Center, has identified a promising gene therapy that could lead to a treatment for DMD. The method delivers a new gene to the body's muscles to replace the defective, disease-causing gene.

As the leading genetic killer of young boys, DMD affects more than 1 in 3,500 boys worldwide and is the most common and lethal form of Muscular Dystrophy. Boys with DMD are usually diagnosed by the age of five, in a wheelchair by age 12, and may be completely paralyzed by their late teens. Historically, most patients with DMD do not live to see adulthood. Duchenne can occur in any family, from any race and from any background.

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Researchers identify promising gene therapy for DMD