Development of a Novel AAV Gene Therapy Cassette with Improved Safety Features and Efficacy in a Mouse Model of Rett Syndrome

Rett syndrome (RTT), caused by loss-of-function mutations in the MECP2 gene, is a neurological disorder characterized by severe impairment of motor and cognitive functions. The aim of this study was to investigate the impact of vector design, dosage, and delivery route on the efficacy and safety of...

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Detalles Bibliográficos
Autores principales: Gadalla, Kamal K.E., Vudhironarit, Thishnapha, Hector, Ralph D., Sinnett, Sarah, Bahey, Noha G., Bailey, Mark E.S., Gray, Steven J., Cobb, Stuart R.
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/PMC5423329/
https://www.ncbi.nlm.nih.gov/pubmed/28497075
http://dx.doi.org/10.1016/j.omtm.2017.04.007

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5423329/
https://www.ncbi.nlm.nih.gov/pubmed/28497075
http://dx.doi.org/10.1016/j.omtm.2017.04.007

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