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Article

Combination therapies for lysosomal storage disease: is the whole greater than the sum of its parts?

Human molecular genetics - 15 Apr 2011

Hawkins-Salsbury Jacqueline A, Reddy Adarsh S, Sands Mark S

Abstract excerpt

Lysosomal storage diseases (LSDs), as a group, are among the most common inherited diseases affecting children. The primary defect is typically a genetic deficiency of one of the lysosomal enzymes, often causing accumulation of undegraded substrates within the lysosome. This accumulation causes numerous secondary effects that contribute to the disease phenotype. Viral-mediated gene therapy (GT) can supply a...

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PMID: 21421999
DOI: 10.1093/hmg/ddr112
PMCID: PMC3095053

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