Transgenic expression of human alpha-L-iduronidase in mouse and characterization of the long term pathophysiology of murine alpha-L-iduronidase deficiency
Mucopolysaccharidosis type I (MPS I) is an autosomal recessive genetic disorder resulting from deficiency of alpha-L-iduronidase (IDUA), a lysosomal hydrolase required in the catabolism of heparan and dermatan sulfate glycosaminoglycans (GAGs). MPS I presents as a clinical spectrum of disease rangin...
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| Format: | Others |
| Language: | English |
| Published: |
2009
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| Online Access: | http://hdl.handle.net/2429/16043 |