Gene therapy for lysosomal storage diseases: progress, challenges and future prospects.

Abstract excerpt

Lysosomal Storage Diseases (LSDs) comprise a group of over fifty inherited metabolic disorders, with their hallmark feature being deficient catabolism and accumulation (storage) of macromolecules in the lysosomes due to genetic deficiency of specific lysosomal enzymes. The combined incidence of LSDs is estimated to be ~1 in 7,000 births. LSD symptoms can vary significantly, primarily due to the nature of the gene...

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