Targeting RNA structure in SMN2 reverses spinal muscular atrophy molecular phenotypes,
Garcia-Lopez, Amparo, Tessaro Francesca, Jonker Hendrik R. A., Wacker Anna, Richter Christian, Comte Arnaud, Berntenis Nikolaos, Schmucki Roland, Hatje Klas, Petermann Olivier, et al.
, Nature Communication, 2018/05/23, Volume 9, Issue 1, p.2032, (2018)