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Hematopoietic Stem Cell Gene Therapy for Storage Disease: Current and New Indications

5 Apr 2017

Abstract excerpt

Lysosomal storage disorders (LSDs) are a broad class of monogenic diseases with an overall incidence of 1:7,000 newborns, due to the defective activity of one or more lysosomal hydrolases or related proteins resulting in storage of un-degraded substrates in the lysosomes. The over 40 different known LSDs share a life-threatening nature, but they are present with extremely variable clinical manifestations,...

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PMID: 28389320
DOI: 10.1016/j.ymthe.2017.03.025

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