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

摘要

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

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Sigma-Aldrich

Monoclonal Anti-MECP2 antibody produced in mouse, clone 4B6, purified immunoglobulin, buffered aqueous solution