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Year Number of Results
1999 1
2000 1
2001 2
2006 1
2008 1
2009 2
2010 2
2011 1
2014 2
2015 1
2016 3
2017 3
2018 3
2019 3
2020 2
2021 5
2024 0

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32 results

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Page 1
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. Among authors: ratni h. Science. 2014 Aug 8;345(6197):688-93. doi: 10.1126/science.1250127. Science. 2014. PMID: 25104390
Discovery of Risdiplam, a Selective Survival of Motor Neuron-2 ( SMN2) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA).
Ratni H, Ebeling M, Baird J, Bendels S, Bylund J, Chen KS, Denk N, Feng Z, Green L, Guerard M, Jablonski P, Jacobsen B, Khwaja O, Kletzl H, Ko CP, Kustermann S, Marquet A, Metzger F, Mueller B, Naryshkin NA, Paushkin SV, Pinard E, Poirier A, Reutlinger M, Weetall M, Zeller A, Zhao X, Mueller L. Ratni H, et al. J Med Chem. 2018 Aug 9;61(15):6501-6517. doi: 10.1021/acs.jmedchem.8b00741. Epub 2018 Jul 25. J Med Chem. 2018. PMID: 30044619
Diversity oriented biosynthesis via accelerated evolution of modular gene clusters.
Wlodek A, Kendrew SG, Coates NJ, Hold A, Pogwizd J, Rudder S, Sheehan LS, Higginbotham SJ, Stanley-Smith AE, Warneck T, Nur-E-Alam M, Radzom M, Martin CJ, Overvoorde L, Samborskyy M, Alt S, Heine D, Carter GT, Graziani EI, Koehn FE, McDonald L, Alanine A, Rodríguez Sarmiento RM, Chao SK, Ratni H, Steward L, Norville IH, Sarkar-Tyson M, Moss SJ, Leadlay PF, Wilkinson B, Gregory MA. Wlodek A, et al. Among authors: ratni h. Nat Commun. 2017 Oct 31;8(1):1206. doi: 10.1038/s41467-017-01344-3. Nat Commun. 2017. PMID: 29089518 Free PMC article.
Binding to SMN2 pre-mRNA-protein complex elicits specificity for small molecule splicing modifiers.
Sivaramakrishnan M, McCarthy KD, Campagne S, Huber S, Meier S, Augustin A, Heckel T, Meistermann H, Hug MN, Birrer P, Moursy A, Khawaja S, Schmucki R, Berntenis N, Giroud N, Golling S, Tzouros M, Banfai B, Duran-Pacheco G, Lamerz J, Hsiu Liu Y, Luebbers T, Ratni H, Ebeling M, Cléry A, Paushkin S, Krainer AR, Allain FH, Metzger F. Sivaramakrishnan M, et al. Among authors: ratni h. Nat Commun. 2017 Nov 14;8(1):1476. doi: 10.1038/s41467-017-01559-4. Nat Commun. 2017. PMID: 29133793 Free PMC article.
32 results