First Clinical Trial of Gene Therapy for Muscular Dystrophy Now Under Way
Date: 7.4.2006
The clinical trial for Duchenne muscular dystrophy (DMD) tests the safety and effectiveness of a therapy that was developed over two decades by scientists at the University of North Carolina at Chapel Hill's School of Medicine and the University of Pittsburgh. In the trial, six boys with DMD will receive replacement genes for an essential muscle protein.
The gene for dystrophin is one of the largest genes in the human body, and miniaturizing it, while retaining the crucial elements of its set of DNA instructions, has been among the greatest challenges to the gene therapy field. The new Biostrophin therapy uses a novel combination of advanced technologies, including a miniaturized replacement dystrophin gene and nano delivery technology called biological nanoparticles. Developed from a virus known as adeno-associated virus (AAV), the nanoparticles are engineered specifically to target and carry the "minidystrophin" gene to muscle cells.
"Source":[ http://www.bio.com/newsfeatures/newsfeatures_research.jhtml?cid=18400004].
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