The Antisense Transcript SMN-AS1 Regulates SMN Expression and Is a Novel Therapeutic Target for Spinal Muscular Atrophy
反义转录本SMN-AS1调控SMN表达，是脊髓性肌萎缩症的新型治疗靶点。
期刊:Neuron
影响因子:15
doi:10.1016/j.neuron.2016.11.033
d'Ydewalle, Constantin; Ramos, Daniel M; Pyles, Noah J; Ng, Shi-Yan; Gorz, Mariusz; Pilato, Celeste M; Ling, Karen; Kong, Lingling; Ward, Amanda J; Rubin, Lee L; Rigo, Frank; Bennett, C Frank; Sumner, Charlotte J
