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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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efficacy
gene therapy
Hurler's syndrome
lysosomal storage disease
MRI,abnormal
mucopolysaccharidoses
safety
stem cell transplantation
treatment of neurologic disorder

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