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Article

Hematopoietic differentiation of induced pluripotent stem cells from patients with mucopolysaccharidosis type I (Hurler syndrome)

30 Oct 2010

Abstract excerpt

Mucopolysaccharidosis type I (MPS IH; Hurler syndrome) is a congenital deficiency of α-L-iduronidase, leading to lysosomal storage of glycosaminoglycans that is ultimately fatal following an insidious onset after birth. Hematopoietic cell transplantation (HCT) is a life-saving measure in MPS IH. However, because a suitable hematopoietic donor is not found for everyone, because HCT is associated with significant...

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Lysosomal Storage Disorders Research
Neurogenetic and Muscular Disorders Research
Pluripotent Stem Cells Research

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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PMID: 21037085
DOI: 10.1182/blood-2010-05-287607

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