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Gene therapy approaches for lysosomal storage disease: next-generation treatment.

ABSTRACT: 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.

SUBMITTER: Byrne BJ

PROVIDER: S-EPMC3413894 | biostudies-literature | 2012 Aug

REPOSITORIES: biostudies-literature

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Gene therapy approaches for lysosomal storage disease: next-generation treatment.

Byrne Barry J BJ Falk Darin J DJ Clément Nathalie N Mah Cathryn S CS

Human gene therapy 20120801 8

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 man ...[more]

PMID: 22794786

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