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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: kothary 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: kothary r. EBioMedicine. 2024 Dec;110:105444. doi: 10.1016/j.ebiom.2024.105444. Epub 2024 Nov 7. EBioMedicine. 2024. PMID: 39515026 Free PMC article.
Identification of the Wnt signal peptide that directs secretion on extracellular vesicles.
Gurriaran-Rodriguez U, Datzkiw D, Radusky LG, Esper M, Javandoost E, Xiao F, Ming H, Fisher S, Marina A, De Repentigny Y, Kothary R, Azkargorta M, Elortza F, Rojas AL, Serrano L, Hierro A, Rudnicki MA. Gurriaran-Rodriguez U, et al. Among authors: kothary r. Sci Adv. 2024 Dec 13;10(50):eado5914. doi: 10.1126/sciadv.ado5914. Epub 2024 Dec 11. Sci Adv. 2024. PMID: 39661666 Free PMC article.
The systemic complexity of a monogenic disease: the molecular network of spinal muscular atrophy.
Tapken I, Schweitzer T, Paganin M, Schüning T, Detering NT, Sharma G, Niesert M, Saffari A, Kuhn D, Glynn A, Cieri F, Santonicola P, Cannet C, Gerstner F, Faller KME, Huang YT, Kothary R, Gillingwater TH, Di Schiavi E, Simon CM, Hensel N, Ziegler A, Viero G, Pich A, Claus P. Tapken I, et al. Among authors: kothary r. Brain. 2024 Aug 26:awae272. doi: 10.1093/brain/awae272. Online ahead of print. Brain. 2024. PMID: 39183150
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: kothary 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.
194 results