Gene therapy approaches for lysosomal storage disease: next-generation treatment.
Lysosomal storage diseases are a group of rare inborn errors of metabolism resulting from deficiency in normal lysosomal function. These diseases are characterized by progressive accumulation of storage material within the lysosomes of affected cells, ultimately leading to cellular dysfunction. Multiple tissues ranging from musculoskeletal and visceral to tissues of the central nervous system are typically involved in disease pathology. Since the advent of enzyme replacement therapy (ERT) to manage some LSDs, general clinical outcomes have significantly improved; however, treatment with infused protein is lifelong and continued disease progression is still evident in patients. Viral gene therapy may provide a viable alternative or adjunctive therapy to current management strategies for LSDs. In this review, we discuss the various viral vector systems that have been developed and some of the strategy designs for the treatment of LSDs.
Department of Pediatrics and Powell Gene Therapy Center, University of Florida, Gainesville, FL 32610, USA. bbyrne@ufl.edu
Barry J
BJ
Byrne
Darin J
DJ
Falk
Nathalie
N
Clément
Cathryn S
CS
Mah
D000818:Animals; D002986:Clinical Trials as Topic; D056947:Enzyme Replacement Therapy; D015316:Genetic Therapy; D005822:Genetic Vectors; D006801:Humans; D016464:Lysosomal Storage Diseases / Q000235:genetics* / Q000628:therapy*