Correction of Clinical Manifestations of Canine Mucopolysaccharidosis I with Neonatal Retroviral Vec...
Correction of Clinical Manifestations of Canine Mucopolysaccharidosis I with Neonatal Retroviral Vector Gene Therapy
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Publisher
United States: Elsevier Inc
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Language
English
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United States: Elsevier Inc
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Contents
Mucopolysaccharidosis I (MPS I) (Hurler syndrome) is due to deficient α-l-iduronidase (IDUA) activity and is the most common of the MPS disorders. Neonatal MPS I dogs were injected intravenously (IV) with a gamma retroviral vector containing a complete long-terminal repeat (LTR) and an internal human α1-antitrypsin (hAAT) promoter upstream of the c...
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Full title
Correction of Clinical Manifestations of Canine Mucopolysaccharidosis I with Neonatal Retroviral Vector Gene Therapy
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TN_cdi_proquest_journals_1792608802
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_journals_1792608802
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ISSN
1525-0016
E-ISSN
1525-0024
DOI
10.1038/sj.mt.6300201
