244. AAV Vector-Mediated Gene Therapy of Mucopolysaccharidosis Type I
244. AAV Vector-Mediated Gene Therapy of Mucopolysaccharidosis Type I
About this item
Full title
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Publisher
Milwaukee: Elsevier Inc
Journal title
Language
English
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Publication information
Publisher
Milwaukee: Elsevier Inc
Subjects
More information
Scope and Contents
Contents
Mucopolysaccharidosis type I (MPSI) is a lysosomal storage disorder caused by loss of activity of the enzyme α-L-iduronidase (IDUA), which is responsible for the catabolism of the glycosaminoglycans dermatan and heparan sulfate. The most severely manifested form of the disease is Hurler syndrome, in which death affects children by the first decade,...
Alternative Titles
Full title
244. AAV Vector-Mediated Gene Therapy of Mucopolysaccharidosis Type I
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Record Identifier
TN_cdi_proquest_journals_1793436878
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_journals_1793436878
Other Identifiers
ISSN
1525-0016
E-ISSN
1525-0024
DOI
10.1016/j.ymthe.2005.06.247
