Intravenous delivery for treatment of mucopolysaccharidosis type I: A comparison of AAV serotypes 9 and rh10

Intravenous delivery for treatment of mucopolysaccharidosis type I: A comparison of AAV serotypes 9 and rh10

Mucopolysaccharidosis type I (MPS I) is an inherited metabolic disorder caused by deficiency of alpha-L-iduronidase (IDUA), resulting in accumulation of heparan and dermatan sulfate glycosaminoglycans (GAGs). Individuals with the most severe form of the disease (Hurler syndrome) suffer from neurodeg...

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Bibliographic Details
Main Authors: Lalitha R. Belur, Kelly M. Podetz-Pedersen, Thuy An Tran, Joshua A. Mesick, Nathaniel M. Singh, Maureen Riedl, Lucy Vulchanova, Karen F. Kozarsky, R. Scott McIvor
Format: Article
Language:English
Published: Elsevier 2020-09-01
Series:Molecular Genetics and Metabolism Reports
Subjects:
Gene therapy
AAV9
AAVrh10
Mucopolysaccharidosis Type I (MPS I)
Iduronidase (IDUA)
Intravenous delivery
Online Access:http://www.sciencedirect.com/science/article/pii/S2214426920300501

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http://www.sciencedirect.com/science/article/pii/S2214426920300501

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