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Early skeletal outcomes after hematopoietic stem and progenitor cell gene therapy for Hurler syndrome

1 May 2024

Abstract excerpt

Mucopolysaccharidosis type I Hurler (MPSIH) is characterized by severe and progressive skeletal dysplasia that is not fully addressed by allogeneic hematopoietic stem cell transplantation (HSCT). Autologous hematopoietic stem progenitor cell-gene therapy (HSPC-GT) provides superior metabolic correction in patients with MPSIH compared with HSCT; however, its ability to affect skeletal manifestations is unknown....

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PMID: 38691622
DOI: 10.1126/scitranslmed.adi8214

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