This is a proposal to study the liposomal preparation of CMV-driven alpha glucosidase or acid maltase (GAA) which is the enzyme deficient in glycogen storage disease type II (GSDII). The investigator will use a GAA knockout mouse to study various infusion regimens and obtain animal data re: the presence of DNA, GAA mRNA and protein. This study may lead to human trials of liposomal packaged DNA for the treatment of acid maltase deficiency. The Core Laboratory was utilized for DNA Isolation, DNA sequencing (manual), oligonucleotide synthesis, and Recombinant DNA techniques.
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