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New Indications for Hematopoietic Stem Cell Gene Therapy in Lysosomal Storage Disorders

13 May 2022

Abstract excerpt

Lysosomal storage disorders (LSDs) are a heterogenous group of disorders due to genetically determined deficits of lysosomal enzymes. The specific molecular mechanism and disease phenotype depends on the type of storage material. Several disorders affect the brain resulting in severe clinical manifestations that substantially impact the expectancy and quality of life. Current treatment modalities for LSDs include...

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PMID: 35646708
DOI: 10.3389/fonc.2022.885639

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