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Commentary and podcast on landmark gene therapy clinical trial for hemophilia B
Date:12/13/2011

se included a self-complementary genome to increase efficiency pioneered by R. Jude Samulski, PhD, Director, Gene Therapy Center, University of North Carolina at Chapel Hill, and the use of a novel AAV capsid from a natural rhesus monkey isolate called AAV8, which was discovered and developed by James M. Wilson, MD, PhD, Editor-in-Chief of Human Gene Therapy and Human Gene Therapy Methods, and Director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia.

Recently published articles in Human Gene Therapy document the development and optimization of the AAV vectors used to deliver the FIX gene in this clinical trial. Two key papers are:

"The successful treatment of patients with hemophilia B with gene therapy is a landmark event representing the culmination of over 20 years of work by many laboratories," says James M. Wilson, MD, PhD. "We are delighted that Human Gene Therapy was a venue for publishing critical aspects of the development of this product."


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Contact: Cathia Falvey
cfalvey@liebertpub.com
914-740-2165
Mary Ann Liebert, Inc./Genetic Engineering News
Source:Eurekalert  

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