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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| Autors principals: | , , , , , , , , , , |
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| Format: | Artigo |
| Idioma: | Inglês |
| Publicat: |
Elsevier
2015-10-01
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| Col·lecció: | Neurobiology of Disease |
| Matèries: | |
| Accés en línia: | http://www.sciencedirect.com/science/article/pii/S0969996115001758 |
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