Gene therapy for lysosomal storage diseases.
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.
Department of Internal Medicine, Washington University School of Medicine, St. Louis, MO 63110, USA. msands@im.wustl.edu
Mark S
MS
Sands
Beverly L
BL
Davidson
D000818:Animals; D018014:Gene Transfer Techniques; D015316:Genetic Therapy; D005822:Genetic Vectors; D006801:Humans; D016464:Lysosomal Storage Diseases / Q000628:therapy*; D008954:Models, Biological