Improved MECP2 Gene Therapy Extends the Survival of MeCP2-Null Mice without Apparent Toxicity after Intracisternal Delivery

Improved MECP2 Gene Therapy Extends the Survival of MeCP2-Null Mice without Apparent Toxicity after Intracisternal Delivery

Intravenous administration of adeno-associated virus serotype 9 (AAV9)/hMECP2 has been shown to extend the lifespan of Mecp2−/y mice, but this delivery route induces liver toxicity in wild-type (WT) mice. To reduce peripheral transgene expression, we explored the safety and efficacy of AAV9/hMECP2 i...

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Bibliographic Details
Main Authors: Sarah E. Sinnett, Ralph D. Hector, Kamal K.E. Gadalla, Clifford Heindel, Daphne Chen, Violeta Zaric, Mark E.S. Bailey, Stuart R. Cobb, Steven J. Gray
Format: Article
Language:English
Published: Elsevier 2017-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
Rett syndrome
MeCP2
cisterna magna
intrathecal
microRNA
AAV
viral vector
Mecp2−/y
Online Access:http://www.sciencedirect.com/science/article/pii/S232905011730061X

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http://www.sciencedirect.com/science/article/pii/S232905011730061X

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