Neonatal Gene Therapy of MPS I Mice by Intravenous Injection of a Lentiviral Vector
Neonatal Gene Therapy of MPS I Mice by Intravenous Injection of a Lentiviral Vector
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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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Scope and Contents
Contents
Mucopolysaccharidosis type I (MPS I) is a lysosomal glycosaminoglycan (GAG) storage disorder caused by deficiency of α-l-iduronidase (IDUA). In this study, we evaluated the potential to perform gene therapy for MPS I by direct in vivo injection of a lentiviral vector, using an IDUA gene knockout murine model. We compared the efficacy in newborn ver...
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Full title
Neonatal Gene Therapy of MPS I Mice by Intravenous Injection of a Lentiviral Vector
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TN_cdi_proquest_miscellaneous_67778052
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_miscellaneous_67778052
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ISSN
1525-0016
E-ISSN
1525-0024
DOI
10.1016/j.ymthe.2004.10.006
