Survival benefit and phenotypic improvement by hamartin gene therapy in a tuberous sclerosis mouse brain model

Survival benefit and phenotypic improvement by hamartin gene therapy in a tuberous sclerosis mouse brain model

We examined the potential benefit of gene therapy in a mouse model of tuberous sclerosis complex (TSC) in which there is embryonic loss of Tsc1 (hamartin) in brain neurons. An adeno-associated virus (AAV) vector (serotype rh8) expressing a tagged form of hamartin was injected into the cerebral ventr...

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Bibliographic Details
Main Authors: Shilpa Prabhakar, Xuan Zhang, June Goto, Sangyeul Han, Charles Lai, Roderick Bronson, Miguel Sena-Esteves, Vijaya Ramesh, Anat Stemmer-Rachamimov, David J. Kwiatkowski, Xandra O. Breakefield
Format: Article
Language:English
Published: Elsevier 2015-10-01
Series:Neurobiology of Disease
Subjects:
Tuberous sclerosis complex
TSC
TSC1
TSC2
Gene therapy
AAV
Online Access:http://www.sciencedirect.com/science/article/pii/S0969996115001758

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http://www.sciencedirect.com/science/article/pii/S0969996115001758

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