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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: 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.
Wnt binding to Coatomer proteins directs secretion on exosomes independently of palmitoylation.
Gurriaran-Rodriguez U, Datzkiw D, Radusky LG, Esper M, Xiao F, Ming H, Fisher S, Rojas MA, De Repentigny Y, Kothary R, Rojas AL, Serrano L, Hierro A, Rudnicki MA. Gurriaran-Rodriguez U, et al. Among authors: kothary r. bioRxiv [Preprint]. 2023 May 30:2023.05.30.542914. doi: 10.1101/2023.05.30.542914. bioRxiv. 2023. PMID: 37398399 Free PMC article. Preprint.
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.
Dysregulation of Tweak and Fn14 in skeletal muscle of spinal muscular atrophy mice.
Meijboom KE, Sutton ER, McCallion E, McFall E, Anthony D, Edwards B, Kubinski S, Tapken I, Bünermann I, Hazell G, Ahlskog N, Claus P, Davies KE, Kothary R, Wood MJA, Bowerman M. Meijboom KE, et al. Among authors: kothary r. Skelet Muscle. 2022 Jul 28;12(1):18. doi: 10.1186/s13395-022-00301-z. Skelet Muscle. 2022. PMID: 35902978 Free PMC article.
264th ENMC International Workshop: Multi-system involvement in spinal muscular atrophy Hoofddorp, the Netherlands, November 19th - 21st 2021.
Detering NT, Zambon A, Hensel N, Kothary R, Swoboda K, Gillingwater TH, Baranello G; Workshop participants; Industry participants. Detering NT, et al. Among authors: kothary r. Neuromuscul Disord. 2022 Aug;32(8):697-705. doi: 10.1016/j.nmd.2022.06.005. Epub 2022 Jun 17. Neuromuscul Disord. 2022. PMID: 35794048 No abstract available.
188 results