Gene therapy for mucopolysaccharidosis

Katherine P. Ponder, Mark E. Haskins

Research output: Contribution to journalReview articlepeer-review

76 Scopus citations


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 can correct disease in liver and spleen, although correction in other organs including the brain requires high enzyme activity in the blood. Alternative approaches are to transduce hematopoietic stem cells, or to inject a vector locally into difficult-to-reach sites such as the brain. Gene therapy holds great promise for providing a long-lasting therapeutic effect for MPS if safety issues can be resolved.

Original languageEnglish
Pages (from-to)1333-1345
Number of pages13
JournalExpert opinion on biological therapy
Issue number9
StatePublished - Sep 2007


  • Gene therapy
  • Glycosaminoglycan
  • Lysosomal storage disease
  • Mucopolysaccharidosis


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