Title : Liver-directed gene therapy corrects cardiovascular lesions in feline mucopolysaccharidosis type I.

Pub. Date : 2014 Oct 14

PMID : 25267637






2 Functional Relationships(s)
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1 Patients with mucopolysaccharidosis type I (MPS I), a genetic deficiency of the lysosomal enzyme alpha-l-iduronidase (IDUA), exhibit accumulation of glycosaminoglycans in tissues, with resulting diverse clinical manifestations including neurological, ocular, skeletal, and cardiac disease. Glycosaminoglycans alpha-L-iduronidase Homo sapiens
2 Patients with mucopolysaccharidosis type I (MPS I), a genetic deficiency of the lysosomal enzyme alpha-l-iduronidase (IDUA), exhibit accumulation of glycosaminoglycans in tissues, with resulting diverse clinical manifestations including neurological, ocular, skeletal, and cardiac disease. Glycosaminoglycans alpha-L-iduronidase Homo sapiens