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Gene therapy for mucopolysaccharidosis

29 Aug 2007

Abstract excerpt

Mucopolysaccharidoses (MPS) are due to deficiencies in activities of lysosomal enzymes that degrade glycosaminoglycans. Some attempts at gene therapy for MPS in animal models have involved intravenous injection of vectors derived from an adeno-associated virus (AAV), adenovirus, retrovirus or a plasmid, which primarily results in expression in liver and secretion of the relevant enzyme into blood. Most vectors...

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PMID: 17727324
DOI: 10.1517/14712598.7.9.1333

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