MUSCLE PROMOTERS --ACID MALTASE DEFICIENCY GENE THERAPY

$0M01FY2000RRNIH

New York University School Of Medicine, New York NY

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Abstract

Inherited deficiency of acid maltase leads to the development of glycogen storage disease type II. There is no treatment or cure for this disease. This is a laboratory protocol to test the three different promotor-enhancers (CMV, creatine kinase, and myosin heavy chain) in vitro and in acid maltase knockout mice in preparation for the development of gene therapy. The Core Laboratory was utilized for DNA Isolation, DNA sequencing (manual), oligonucleotide synthesis, and Recombinant DNA techniques.

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