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The DHODH inhibitor PTC299 arrests SARS-CoV-2 replication and suppresses induction of inflammatory cytokines.
Luban J, Sattler RA, Mühlberger E, Graci JD, Cao L, Weetall M, Trotta C, Colacino JM, Bavari S, Strambio-De-Castillia C, Suder EL, Wang Y, Soloveva V, Cintron-Lue K, Naryshkin NA, Pykett M, Welch EM, O'Keefe K, Kong R, Goodwin E, Jacobson A, Paessler S, Peltz SW. Luban J, et al. Among authors: naryshkin na. Virus Res. 2021 Jan 15;292:198246. doi: 10.1016/j.virusres.2020.198246. Epub 2020 Nov 26. Virus Res. 2021. PMID: 33249060 Free PMC article.
Nonsense suppression activity of PTC124 (ataluren).
Peltz SW, Welch EM, Jacobson A, Trotta CR, Naryshkin N, Sweeney HL, Bedwell DM. Peltz SW, et al. Proc Natl Acad Sci U S A. 2009 Jun 23;106(25):E64; author reply E65. doi: 10.1073/pnas.0901936106. Epub 2009 Jun 8. Proc Natl Acad Sci U S A. 2009. PMID: 19506240 Free PMC article. No abstract available.
Motor neuron disease. SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy.
Naryshkin NA, Weetall M, Dakka A, Narasimhan J, Zhao X, Feng Z, Ling KK, Karp GM, Qi H, Woll MG, Chen G, Zhang N, Gabbeta V, Vazirani P, Bhattacharyya A, Furia B, Risher N, Sheedy J, Kong R, Ma J, Turpoff A, Lee CS, Zhang X, Moon YC, Trifillis P, Welch EM, Colacino JM, Babiak J, Almstead NG, Peltz SW, Eng LA, Chen KS, Mull JL, Lynes MS, Rubin LL, Fontoura P, Santarelli L, Haehnke D, McCarthy KD, Schmucki R, Ebeling M, Sivaramakrishnan M, Ko CP, Paushkin SV, Ratni H, Gerlach I, Ghosh A, Metzger F. Naryshkin NA, et al. Science. 2014 Aug 8;345(6197):688-93. doi: 10.1126/science.1250127. Science. 2014. PMID: 25104390
Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy.
Zhao X, Feng Z, Ling KK, Mollin A, Sheedy J, Yeh S, Petruska J, Narasimhan J, Dakka A, Welch EM, Karp G, Chen KS, Metzger F, Ratni H, Lotti F, Tisdale S, Naryshkin NA, Pellizzoni L, Paushkin S, Ko CP, Weetall M. Zhao X, et al. Among authors: naryshkin na. Hum Mol Genet. 2016 May 15;25(10):1885-1899. doi: 10.1093/hmg/ddw062. Epub 2016 Feb 29. Hum Mol Genet. 2016. PMID: 26931466 Free PMC article.
Discovery and Optimization of Small Molecule Splicing Modifiers of Survival Motor Neuron 2 as a Treatment for Spinal Muscular Atrophy.
Woll MG, Qi H, Turpoff A, Zhang N, Zhang X, Chen G, Li C, Huang S, Yang T, Moon YC, Lee CS, Choi S, Almstead NG, Naryshkin NA, Dakka A, Narasimhan J, Gabbeta V, Welch E, Zhao X, Risher N, Sheedy J, Weetall M, Karp GM. Woll MG, et al. Among authors: naryshkin na. J Med Chem. 2016 Jul 14;59(13):6070-85. doi: 10.1021/acs.jmedchem.6b00460. Epub 2016 Jul 6. J Med Chem. 2016. PMID: 27299569
The DHODH Inhibitor PTC299 Arrests SARS-CoV-2 Replication and Suppresses Induction of Inflammatory Cytokines.
Luban J, Sattler R, Mühlberger E, Graci JD, Cao L, Weetall M, Trotta C, Colacino JM, Bavari S, Strambio-De-Castillia C, Suder EL, Wang Y, Soloveva V, Cintron-Lue K, Naryshkin NA, Pykett M, Welch EM, O'Keefe K, Kong R, Goodwin E, Jacobson A, Paessler S, Peltz S. Luban J, et al. Among authors: naryshkin na. bioRxiv [Preprint]. 2020 Aug 5:2020.08.05.238394. doi: 10.1101/2020.08.05.238394. bioRxiv. 2020. PMID: 32793904 Free PMC article. Updated. Preprint.
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: naryshkin na. Nat Commun. 2021 Dec 15;12(1):7299. doi: 10.1038/s41467-021-27157-z. Nat Commun. 2021. PMID: 34911927 Free PMC article.
25 results