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Page 1
Base editing as a genetic treatment for spinal muscular atrophy.
Alves CRR, Ha LL, Yaworski R, 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: yaworski r. bioRxiv [Preprint]. 2023 Jan 21:2023.01.20.524978. doi: 10.1101/2023.01.20.524978. bioRxiv. 2023. PMID: 36711797 Free PMC article. Preprint.
Liver SMN restoration rescues the Smn2B/- mouse model of spinal muscular atrophy.
Sutton ER, Beauvais A, Yaworski R, De Repentigny Y, Reilly A, Alves de Almeida MM, Deguise MO, Poulin KL, Parks RJ, Schneider BL, Kothary R. Sutton ER, et al. Among authors: yaworski r. EBioMedicine. 2024 Dec;110:105444. doi: 10.1016/j.ebiom.2024.105444. Epub 2024 Nov 7. EBioMedicine. 2024. PMID: 39515026 Free PMC article.
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: yaworski r. 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.
Hepatic Choline Transport Is Inhibited During Fatty Acid-Induced Lipotoxicity and Obesity.
O'Dwyer C, Yaworski R, Katsumura S, Ghorbani P, Gobeil Odai K, Nunes JRC, LeBlond ND, Sanjana S, Smith TTK, Han S, Margison KD, Alain T, Morita M, Fullerton MD. O'Dwyer C, et al. Among authors: yaworski r. Hepatol Commun. 2020 Apr 10;4(6):876-889. doi: 10.1002/hep4.1516. eCollection 2020 Jun. Hepatol Commun. 2020. PMID: 32490323 Free PMC article.
17 results