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Optimized Base Editing Reagents Yield More Potent Genetic Correction in a Mouse Model of Alpha-1 Antitrypsin Deficiency

Michael S. Packer,Dominique Leboeuf,Lo-I Cheng,Raymond Yang,Jeremy Decker,Monique Otero,Luis A. Barrera,Brian J. Cafferty,Valerie J. Winton,Yvonne Aratyn-Schaus,Sarah Smith,Giuseppe Ciaramella,Francine M. Gregoire

MOLECULAR THERAPY（2022）

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