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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Dettagli Bibliografici
Pubblicato in:Neurobiol Dis
Autori principali: Prabhakar, Shilpa, Zhang, Xuan, Goto, June, Han, Sangyeul, Lai, Charles, Bronson, Roderick, Sena-Esteves, Miguel, Ramesh, Vijaya, Stemmer-Rachamimov, Anat, Kwiatkowski, David J., Breakefield, Xandra O.
Natura: Artigo
Lingua:Inglês
Pubblicazione: 2015
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Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC5070799/
https://ncbi.nlm.nih.gov/pubmed/26019056
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.nbd.2015.04.018
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