PAMless Base Editors as a Genetic Treatment for Spinal Muscular Atrophy
Christiano R. R. Alves, Ha L. Leillani,Rebecca Yaworski,Cicera R. Lazzarotto, Kathleen Christie,Aoife Reilly,Ariane Beauvais,Roman M. Doll, Demitri de la Cruz, Casey A. Maguire, Kathryn J. Swoboda,Shengdar Q. Tsai, Rashmikant Kothary, Benjamin P. Kleinstiver MOLECULAR THERAPY(2023)
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