Ex vivo gene therapy for lysosomal storage disorders: future perspectives.

Expert opinion on biological therapy（2023）

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摘要

Clinical trials are already ongoing, and soon approved therapies for LSD based on ex vivo gene therapy approaches should reach the market.

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关键词

Genome editing,Lysosomal storage disorders,adeno-associated vectors,ex vivo gene therapy,hematopoietic stem cells,lentiviral vectors

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