Gene Therapy
Researchers at University of Texas, Department of Pediatrics target gene therapy
Published in Genetics and Environmental Business Week, November 23rd, 2006
Research findings, "Hyaluronidase increases the biodistribution of acid alpha-1,4 glucosidase in the muscle of Pompe disease mice: an approach to enhance the efficacy of enzyme replacement therapy," are discussed in a new report. According to recent research from the United States, "Pompe disease (glycogen storage disease type II) is a glycogen storage disease caused by a deficiency of the lysosomal enzyme, acid maltase/acid alpha-1,4 glucosidase (GAA). Deficiency of the enzyme leads primarily to intra-lysosomal glycogen accumulation, primarily in cardiac and skeletal muscles, due to the inability of converting glycogen into glucose."
"Enzyme replacement therapy (ERT)...
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