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Cell and Gene-Based Therapies for the Lysosomal Storage Diseases

28 Mar 2006

Abstract excerpt

Lysosomal storage disorders (LSD) are a group of approximately 40 genetic diseases that are caused by the deficiency of one or more lysosomal enzymes. The incidence of LSD is estimated to be approximately 1 in 7500 live births, which makes this one of the more prevalent groups of genetic diseases in humans. The loss in enzymatic activity leads to the accumulation of undegraded substrates within lysosomes,...

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PMID: 16611044
DOI: 10.2174/156652306776359522

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