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