Global ETD Search
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Showing 1 to 1 of 1 for “"Mucoploysaccharidosis"”.
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Molecular therapy for mucopolysaccharidosis Type I
Mucopolysaccharidosis type I (MPS I) is caused by deficiency of the lysosomal hydrolase alpha-L-iduronidase (IDUA). IDUA is a required component of the step-wise degradative pathway responsible for the catabolism of the glycosaminoglycans (GAGs) heparan sulfate and dermatan sulfate. As a result, …