Ontology highlight
ABSTRACT:
SUBMITTER: Tolar J
PROVIDER: S-EPMC3035077 | biostudies-literature | 2011 Jan
REPOSITORIES: biostudies-literature
Tolar Jakub J Park In-Hyun IH Xia Lily L Lees Chris J CJ Peacock Brandon B Webber Beau B McElmurry Ron T RT Eide Cindy R CR Orchard Paul J PJ Kyba Michael M Osborn Mark J MJ Lund Troy C TC Wagner John E JE Daley George Q GQ Blazar Bruce R BR
Blood 20101029 3
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 morbidity and mortality, and because there is no known benefit of immune reaction bet ...[more]