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University of Pennsylvania - The Institute for Human Gene Therapy
Limb Girdle Muscular Dystrophy Clinical Trial

Limb Girdle Muscular Dystrophy
On September 2, 1999, the Muscular Dystrophy Association announced the start of a new clinical trial - the first time any form of gene therapy has been tried in a muscle disease. The Institute for Human Gene Therapy at the University of Pennsylvania and the Ohio State University Medical Center are conducting this Phase I clinical trial to test the safety of muscle-directed gene therapy for the Limb Girdle muscular dystrophies (LGMD) caused by defects in sarcoglycan genes.

Limb Girdle Muscular Dystrophy is a form of muscular dystrophy affecting tens of thousands of Americans, and many more worldwide. This Phase I trial will focus on a subset of LGMD, which consists of deficiencies in one of four sarcoglycan or membrane proteins that work in muscle cells.

The main objective of this preliminary, Phase I trial is to study the safety of the procedure and to determine a safe dose of the adeno-associated virus (AAV) vector delivered via needle injection to the extensor digitorum brevis (a muscle in the foot). Results will be used in subsequent trials of efficacy for limb girdle muscular dystrophy patients with one of the sarcoglycan deficiencies. Secondary objectives of the trial are to assess the efficiency of gene transfer after vector administration, and to measure cellular immune responses.

The study is based on research conducted in recent years by Dr. Hansell Stedman and others at the Institute for Human Gene Therapy, under the direction of Dr. James Wilson. Results of their research, using a naturally-occurring hamster model of the disease, were published in the cover story of the April 1999 issue of Nature Medicine (5, 439-443) and announced earlier this year. (See Press Release.)

The Institute for Human Gene Therapy has established a Clinical Trials Unit for this study, which can be reached by calling 215-614-0932. Callers will receive follow-up correspondence in the form of a letter as an initial contact.

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