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Article

Neonatal Gene Therapy of MPS I Mice by Intravenous Injection of a Lentiviral Vector

25 Jan 2005

Abstract excerpt

Mucopolysaccharidosis type I (MPS I) is a lysosomal glycosaminoglycan (GAG) storage disorder caused by deficiency of alpha-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 versus young adult MPS I mice of a single intravenous injection...

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PMID: 15851016
DOI: 10.1016/j.ymthe.2004.10.006

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