Delivery of the 7-dehydrocholesterol reductase gene to the central nervous system using adeno-associated virus vector in a mouse model of Smith-Lemli-Opitz Syndrome

Delivery of the 7-dehydrocholesterol reductase gene to the central nervous system using adeno-associated virus vector in a mouse model of Smith-Lemli-Opitz Syndrome

Smith Lemli Opitz syndrome (SLOS) is an inherited malformation and mental retardation metabolic disorder with no cure. Mutations in the last enzyme of the cholesterol biosynthetic pathway, 7-dehydrocholesterol reductase (DHCR7), lead to cholesterol insufficiency and accumulation of its dehyrdocholes...

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Bibliographic Details
Main Authors: Saloni Pasta, Omoye Akhile, Dorothy Tabron, Flora Ting, Cedric Shackleton, Gordon Watson
Format: Article
Language:English
Published: Elsevier 2015-09-01
Series:Molecular Genetics and Metabolism Reports
Subjects:
Smith Lemli Opitz syndrome (SLOS)
7-dehydrocholesterol reductase (DHCR7)
Central nervous system (CNS)
Intrathecal
AAV
Gene therapy
Online Access:http://www.sciencedirect.com/science/article/pii/S2214426915300240

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http://www.sciencedirect.com/science/article/pii/S2214426915300240

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