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. bioRxiv. 2023 Jan 21:2023.01.20.524978. doi: 10.1101/2023.01.20.524978. Preprint. PMID: 36711797 Free PMC article.
Central and peripheral delivered AAV9-SMN are both efficient but target different pathomechanisms in a mouse model of spinal muscular atrophy. Reilly A, Deguise MO, Beauvais A, Yaworski R, Thebault S, Tessier DR, Tabard-Cossa V, Hensel N, Schneider BL, Kothary R. Gene Ther. 2022 Sep;29(9):544-554. doi: 10.1038/s41434-022-00338-1. Epub 2022 Apr 25. PMID: 35462564
SMN Depleted Mice Offer a Robust and Rapid Onset Model of Nonalcoholic Fatty Liver Disease. Deguise MO, Pileggi C, De Repentigny Y, Beauvais A, Tierney A, Chehade L, Michaud J, Llavero-Hurtado M, Lamont D, Atrih A, Wishart TM, Gillingwater TH, Schneider BL, Harper ME, Parson SH, Kothary R. Cell Mol Gastroenterol Hepatol. 2021;12(1):354-377.e3. doi: 10.1016/j.jcmgh.2021.01.019. Epub 2021 Feb 2. PMID: 33545428 Free PMC article.
MiRNA-145-5p prevents differentiation of oligodendrocyte progenitor cells by regulating expression of myelin gene regulatory factor. Kornfeld SF, Cummings SE, Fathi S, Bonin SR, Kothary R. J Cell Physiol. 2021 Feb;236(2):997-1012. doi: 10.1002/jcp.29910. Epub 2020 Jun 30. PMID: 32602617
Inhibitory milieu at the multiple sclerosis lesion site and the challenges for remyelination. Galloway DA, Gowing E, Setayeshgar S, Kothary R. Glia. 2020 May;68(5):859-877. doi: 10.1002/glia.23711. Epub 2019 Aug 23. PMID: 31441132 Review.
