Liver-directed neonatal gene therapy prevents cardiac, bone, ear, and eye disease in mucopolysacchar...
Liver-directed neonatal gene therapy prevents cardiac, bone, ear, and eye disease in mucopolysaccharidosis I mice
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Publisher
United States: Elsevier Inc
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Language
English
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Publisher
United States: Elsevier Inc
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Mucopolysaccharidosis I (MPS I) due to deficient α-l-iduronidase (IDUA) activity results in accumulation of glycosaminoglycans in many cells. Gene therapy could program liver to secrete enzyme with mannose 6-phosphate (M6P), and enzyme in blood could be taken up by other cells via the M6P receptor. Newborn MPS I mice were injected with 109 (high do...
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Full title
Liver-directed neonatal gene therapy prevents cardiac, bone, ear, and eye disease in mucopolysaccharidosis I mice
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TN_cdi_proquest_miscellaneous_67336386
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_miscellaneous_67336386
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ISSN
1525-0016
E-ISSN
1525-0024
DOI
10.1016/j.ymthe.2004.08.027
