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Comparison of pharmaceutical properties and biological activities of prednisolone, deflazacort, and vamorolone in DMD disease models.
Liu G, Lipari P, Mollin A, Jung S, Teplova I, Li W, Ying L, More V, Lennox W, Yeh S, McGann E, Moon YC, Rice C, Huarte E, Gruszka B, Ray B, Goodwin E, Buckendahl P, Yurkow E, Braughton B, Narasimhan J, Welch E, Voronin G, Weetall M. Liu G, et al. Among authors: narasimhan j. Hum Mol Genet. 2024 Jan 20;33(3):211-223. doi: 10.1093/hmg/ddad173. Hum Mol Genet. 2024. PMID: 37819629 Free PMC article.
Development of an oral treatment that rescues gait ataxia and retinal degeneration in a phenotypic mouse model of familial dysautonomia.
Morini E, Chekuri A, Logan EM, Bolduc JM, Kirchner EG, Salani M, Krauson AJ, Narasimhan J, Gabbeta V, Grover S, Dakka A, Mollin A, Jung SP, Zhao X, Zhang N, Zhang S, Arnold M, Woll MG, Naryshkin NA, Weetall M, Slaugenhaupt SA. Morini E, et al. Among authors: narasimhan j. Am J Hum Genet. 2023 Mar 2;110(3):531-547. doi: 10.1016/j.ajhg.2023.01.019. Epub 2023 Feb 20. Am J Hum Genet. 2023. PMID: 36809767 Free PMC article.
Reduction of retinal ganglion cell death in mouse models of familial dysautonomia using AAV-mediated gene therapy and splicing modulators.
Schultz A, Cheng SY, Kirchner E, Costello S, Miettinen H, Chaverra M, King C, George L, Zhao X, Narasimhan J, Weetall M, Slaugenhaupt S, Morini E, Punzo C, Lefcort F. Schultz A, et al. Among authors: narasimhan j. Sci Rep. 2023 Oct 30;13(1):18600. doi: 10.1038/s41598-023-45376-w. Sci Rep. 2023. PMID: 37903840 Free PMC article.
Reduction of retinal ganglion cell death in mouse models of familial dysautonomia using AAV-mediated gene therapy and splicing modulators.
Schultz A, Cheng SY, Kirchner E, Costello S, Miettinen H, Chaverra M, King C, George L, Zhao X, Narasimhan J, Weetall M, Slaugenhaupt S, Morini E, Punzo C, Lefcort F. Schultz A, et al. Among authors: narasimhan j. bioRxiv [Preprint]. 2023 May 24:2023.05.22.541535. doi: 10.1101/2023.05.22.541535. bioRxiv. 2023. PMID: 37293016 Free PMC article. Updated. Preprint.
Ribonucleotide reductase, a novel drug target for gonorrhea.
Narasimhan J, Letinski S, Jung SP, Gerasyuto A, Wang J, Arnold M, Chen G, Hedrick J, Dumble M, Ravichandran K, Levitz T, Cui C, Drennan CL, Stubbe J, Karp G, Branstrom A. Narasimhan J, et al. Elife. 2022 Feb 9;11:e67447. doi: 10.7554/eLife.67447. Elife. 2022. PMID: 35137690 Free PMC article.
Small molecule splicing modifiers with systemic HTT-lowering activity.
Bhattacharyya A, Trotta CR, Narasimhan J, Wiedinger KJ, Li W, Effenberger KA, Woll MG, Jani MB, Risher N, Yeh S, Cheng Y, Sydorenko N, Moon YC, Karp GM, Weetall M, Dakka A, Gabbeta V, Naryshkin NA, Graci JD, Tripodi T Jr, Southwell A, Hayden M, Colacino JM, Peltz SW. Bhattacharyya A, et al. Among authors: narasimhan j. Nat Commun. 2021 Dec 15;12(1):7299. doi: 10.1038/s41467-021-27157-z. Nat Commun. 2021. PMID: 34911927 Free PMC article.
41 results