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Enzyme Replacement Therapy in Mucopolysaccharidosis I: Altered Distribution and Targeting of α-l-Iduronidase in Immunized Rats

✍ Scribed by Chris T. Turner; John J. Hopwood; Doug A. Brooks

Book ID
115639661
Publisher
Elsevier Science
Year
2000
Tongue
English
Weight
90 KB
Volume
69
Category
Article
ISSN
1096-7192

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Mucopolysaccharidosis type I (MPS I) is an autosomal recessive lysosomal storage disorder caused by a deficiency of alpha-L-iduronidase (IDUA). Mutations in the gene are responsible for the enzyme deficiency, which leads to the intralysosomal storage of the partially degraded glycosaminoglycans derm