January « 2010 « Health Headlines Explained™

Gene therapy for Pompe disease

A recent article in the journal of Molecular Therapy, representing work from the University of Florida, indicated a possible gene therapy treatment for Pompe disease. Pompe disease results from a mutation in the gene that encodes the enzyme, acid alpha-glucosidase (GAA), which releases glucose from glycogen. Glycogen consists of repeating monomers of glucose and is thus [...]

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