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Optimization of Base Editors for the Functional Correction of SMN2 As a Treatment for Spinal Muscular Atrophy

Christiano R. R. Alves,Leillani L. Ha,Rebecca Yaworski,Emma R. Sutton,Cicera R. Lazzarotto,Kathleen A. Christie,Aoife Reilly,Ariane Beauvais,Roman M. Doll,Demitri de la Cruz,Casey A. Maguire,Kathryn J. Swoboda,Shengdar Q. Tsai,Rashmi Kothary,Benjamin P. Kleinstiver

NATURE BIOMEDICAL ENGINEERING（2024）

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Key words
Spinal Muscular Atrophy,SMN2,Multiplex Genome Editing,Survival Motor Neuron Protein
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