Targeting RNA structure in SMN2 reverses spinal muscular atrophy molecular phenotypes.
靶向 SMN2 中的 RNA 结构可逆转脊髓性肌萎缩症的分子表型
期刊:Nature Communications
影响因子:15.7
doi:10.1038/s41467-018-04110-1
Garcia-Lopez Amparo, Tessaro Francesca, Jonker Hendrik R A, Wacker Anna, Richter Christian, Comte Arnaud, Berntenis Nikolaos, Schmucki Roland, Hatje Klas, Petermann Olivier, Chiriano Gianpaolo, Perozzo Remo, Sciarra Daniel, Konieczny Piotr, Faustino Ignacio, Fournet Guy, Orozco Modesto, Artero Ruben, Metzger Friedrich, Ebeling Martin, Goekjian Peter, Joseph Benoît, Schwalbe Harald, Scapozza Leonardo
