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Optimization of base editors for the functional correction of SMN2 as a treatment for spinal muscular atrophy.

Document Type

Article

Author

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

Source

Nature Biomedical Engineering; Feb2024, Vol. 8 Issue 2, p118-131, 14p

Subject

Language

ISSN

2157846X

Online Access

Web of Science JCR 저널정보 Scopus Find it@PNU

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