Direct gene transfer to the CNS prevents emergence of neurologic disease in a murine model of mucopolysaccharidosis type I

Direct gene transfer to the CNS prevents emergence of neurologic disease in a murine model of mucopolysaccharidosis type I

The mucopolysaccharidoses (MPSs) are a group of 11 storage diseases caused by disruptions in glycosaminoglycan (GAG) catabolism, leading to their accumulation in lysosomes. Resultant multisystemic disease is manifested by growth delay, hepatosplenomegaly, skeletal dysplasias, cardiopulmonary obstruc...

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Bibliographic Details
Main Authors: Daniel A. Wolf, Andrew W. Lenander, Zhenhong Nan, Lalitha R. Belur, Chester B. Whitley, Pankaj Gupta, Walter C. Low, R. Scott McIvor
Format: Article
Language:English
Published: Elsevier 2011-07-01
Series:Neurobiology of Disease
Subjects:
Mucopolysaccharidosis
Hurler syndrome
Lysosomal storage disease
Iduronidase
AAV
Gene transfer
Online Access:http://www.sciencedirect.com/science/article/pii/S0969996111000696

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http://www.sciencedirect.com/science/article/pii/S0969996111000696

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