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Optimization of base editors for the functional correction of SMN2 as a treatment for spinal muscular atrophy.
Alves CRR, Ha LL, Yaworski R, Sutton ER, Lazzarotto CR, Christie KA, Reilly A, Beauvais A, Doll RM, de la Cruz D, Maguire CA, Swoboda KJ, Tsai SQ, Kothary R, Kleinstiver BP. Alves CRR, et al. Among authors: kleinstiver bp. Nat Biomed Eng. 2024 Feb;8(2):118-131. doi: 10.1038/s41551-023-01132-z. Epub 2023 Dec 6. Nat Biomed Eng. 2024. PMID: 38057426 Free PMC article.
Engineered CRISPR-Cas9 nucleases with altered PAM specificities.
Kleinstiver BP, Prew MS, Tsai SQ, Topkar VV, Nguyen NT, Zheng Z, Gonzales AP, Li Z, Peterson RT, Yeh JR, Aryee MJ, Joung JK. Kleinstiver BP, et al. Nature. 2015 Jul 23;523(7561):481-5. doi: 10.1038/nature14592. Epub 2015 Jun 22. Nature. 2015. PMID: 26098369 Free PMC article.
82 results