Gene therapy for lysosomal storage diseases

Mol Ther. 2006 May;13(5):839-49. doi: 10.1016/j.ymthe.2006.01.006. Epub 2006 Mar 20.


Lysosomal storage diseases (LSDs) comprise a diverse group of monogenetic disorders with complex clinical phenotypes that include both systemic and central nervous system pathologies. In recent years, the identification or development of mouse models recapitulating the clinical course of the LSDs has been instrumental in evaluating therapeutic strategies. Here, we review the various gene replacement strategies for target organs affected in many LSDs and describe briefly the various vector systems employed to test how best to accomplish long-lasting therapies for these fatal disorders.

Publication types

  • Research Support, N.I.H., Extramural
  • Review

MeSH terms

  • Animals
  • Gene Transfer Techniques
  • Genetic Therapy*
  • Genetic Vectors
  • Humans
  • Lysosomal Storage Diseases / therapy*
  • Models, Biological