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

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

Molecular therapy. Methods & clinical development (2017-05-13)
Kamal K E Gadalla, Thishnapha Vudhironarit, Ralph D Hector, Sarah Sinnett, Noha G Bahey, Mark E S Bailey, Steven J Gray, Stuart R Cobb
PMID28497075
ABSTRACT

Rett syndrome (RTT), caused by loss-of-function mutations in the