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...

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Detalles Bibliográficos
Autores principales: Sinnett, Sarah E., Hector, Ralph D., Gadalla, Kamal K.E., Heindel, Clifford, Chen, Daphne, Zaric, Violeta, Bailey, Mark E.S., Cobb, Stuart R., Gray, Steven J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2017
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5424572/
https://www.ncbi.nlm.nih.gov/pubmed/28497072
http://dx.doi.org/10.1016/j.omtm.2017.04.006

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