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Hematopoietic Stem - and Progenitor-Cell Gene Therapy for Hurler Syndrome NEJM 385:1929-1940, Gentner, B.,et al, 2021 See this aricle in Pubmed Article Abstract The delivery of HSPC gene therapy in patients with MPSIH resulted in extensive metabolic correction in peripheral tissues and the central nervous system.

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(click to filter results - removes previous filter) efficacy gene therapy Hurler's syndrome lysosomal storage disease MRI,abnormal mucopolysaccharidoses safety stem cell transplantation treatment of neurologic disorder

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