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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: ebeling m. Science. 2014 Aug 8;345(6197):688-93. doi: 10.1126/science.1250127. Science. 2014. PMID: 25104390
Cargo Delivery into the Brain by in vivo identified Transport Peptides.
Urich E, Schmucki R, Ruderisch N, Kitas E, Certa U, Jacobsen H, Schweitzer C, Bergadano A, Ebeling M, Loetscher H, Freskgård PO. Urich E, et al. Among authors: ebeling m. Sci Rep. 2015 Sep 28;5:14104. doi: 10.1038/srep14104. Sci Rep. 2015. PMID: 26411801 Free PMC article.
Functional analysis and transcriptional output of the Göttingen minipig genome.
Heckel T, Schmucki R, Berrera M, Ringshandl S, Badi L, Steiner G, Ravon M, Küng E, Kuhn B, Kratochwil NA, Schmitt G, Kiialainen A, Nowaczyk C, Daff H, Khan AP, Lekolool I, Pelle R, Okoth E, Bishop R, Daubenberger C, Ebeling M, Certa U. Heckel T, et al. Among authors: ebeling m. BMC Genomics. 2015 Nov 14;16:932. doi: 10.1186/s12864-015-2119-7. BMC Genomics. 2015. PMID: 26573612 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: ebeling m. Nat Commun. 2017 Nov 14;8(1):1476. doi: 10.1038/s41467-017-01559-4. Nat Commun. 2017. PMID: 29133793 Free PMC article.
Targeting RNA structure in SMN2 reverses spinal muscular atrophy molecular phenotypes.
Garcia-Lopez A, Tessaro F, Jonker HRA, Wacker A, Richter C, Comte A, Berntenis N, Schmucki R, Hatje K, Petermann O, Chiriano G, Perozzo R, Sciarra D, Konieczny P, Faustino I, Fournet G, Orozco M, Artero R, Metzger F, Ebeling M, Goekjian P, Joseph B, Schwalbe H, Scapozza L. Garcia-Lopez A, et al. Among authors: ebeling m. Nat Commun. 2018 May 23;9(1):2032. doi: 10.1038/s41467-018-04110-1. Nat Commun. 2018. PMID: 29795225 Free PMC article.
267 results