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Gene therapy for lysosomal storage disorders: a good start

24 Nov 2015

Abstract excerpt

Lysosomal storage disorders (LSDs) are a heterogeneous group of inherited diseases with a collective frequency of ∼1 in 7000 births, resulting from the deficiency in one or more enzymes or transporters that normally reside within the lysosomes. Pathology results from the progressive accumulation of uncleaved lipids, glycoproteins and/or glycosaminoglycans in the lysosomes and secondary damages that affect the...

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PMID: 26604151
DOI: 10.1093/hmg/ddv457

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