- 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
RESUMEN
Rett syndrome (RTT), caused by loss-of-function mutations in the
MATERIALES
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Sigma-Aldrich
Monoclonal Anti-MECP2 antibody produced in mouse, clone 4B6, purified immunoglobulin, buffered aqueous solution