Development of a Novel AAV Gene Therapy Cassette with Improved Safety Features and Efficacy in a Mouse Model of Rett Syndrome

Development of a Novel AAV Gene Therapy Cassette with Improved Safety Features and Efficacy in a Mouse Model of Rett Syndrome

Rett syndrome (RTT), caused by loss-of-function mutations in the MECP2 gene, is a neurological disorder characterized by severe impairment of motor and cognitive functions. The aim of this study was to investigate the impact of vector design, dosage, and delivery route on the efficacy and safety of...

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Bibliographic Details
Main Authors: Kamal K.E. Gadalla, Thishnapha Vudhironarit, Ralph D. Hector, Sarah Sinnett, Noha G. Bahey, Mark E.S. Bailey, Steven J. Gray, Stuart R. Cobb
Format: Article
Language:English
Published: Elsevier 2017-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
Rett syndrome. MECP2
adeno-associated virus
gene therapy
neurodevelopmental disorder
autism
vector
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050117300621

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http://www.sciencedirect.com/science/article/pii/S2329050117300621

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