Elements of lentiviral vector design toward gene therapy for treating mucopolysaccharidosis I

Elements of lentiviral vector design toward gene therapy for treating mucopolysaccharidosis I

Mucopolysaccharidosis type I (MPS I) is a lysosomal disease caused by α-l-iduronidase (IDUA) deficiency and accumulation of glycosaminoglycans (GAG). Lentiviral vector encoding correct IDUA cDNA could be used for treating MPS I. To optimize the lentiviral vector design, 9 constructs were designed by...

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Bibliographic Details
Main Authors: Li Ou, Michael J. Przybilla, Brenda L. Koniar, Chester B. Whitley
Format: Article
Language:English
Published: Elsevier 2016-09-01
Series:Molecular Genetics and Metabolism Reports
Subjects:
Lysosomal disease
Hurler syndrome
Mucopolysaccharidosis
Lentivirus
Gene therapy
Online Access:http://www.sciencedirect.com/science/article/pii/S2214426915300549

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http://www.sciencedirect.com/science/article/pii/S2214426915300549

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