SMN2 splice modulators enhance U1–pre-mRNA association and rescue SMA mice James Palacino , Susanne E Swalley , Cheng Song , Atwood K Cheung , Lei Shu , Xiaolu Zhang , Mailin Van Hoosear , Youngah Shin , Donovan N Chin , Caroline Gubser Keller , Martin Beibel , Nicole A Renaud , Thomas M Smith , Michael Salcius , Xiaoying Shi , Marc Hild , Rebecca Servais , Monish Jain , Lin Deng , Caroline Bullock , Michael McLellan , Sven Schuierer , Leo Murphy , Marcel J J Blommers , Cecile Blaustein , Frada Berenshteyn , Arnaud Lacoste , Jason R Thomas , Guglielmo Roma , Gregory A Michaud , Brian S Tseng , Jeffery A Porter , Vic E Myer , John A Tallarico , Lawrence G Hamann , Daniel Curtis , Mark C Fishman , William F Dietrich , Natalie A Dales , Rajeev Sivasankaran NATURE CHEMICAL BIOLOGY（2015）
摘要
high-throughput screen identified a small molecule that promoted inclusion of SMN2 exon 7, increased SMN2 protein levels and extended survival in a SMA mouse model through stabilization of the interaction between SMN2 pre-mRNA and U1 snRNP complex.
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关键词
small molecules, rna splicing
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