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

Kamal K.E. Gadalla, Thishnapha Vudhironarit, Ralph D. Hector, Sarah Sinnett, Noha G. Bahey, Mark E.S. Bailey, Steven J. Gray, Stuart R. Cobb

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40 Scopus citations

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Medicine & Life Sciences