Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy
将反义寡核苷酸递送至小鼠中枢神经系统可缓解严重脊髓性肌萎缩症的症状。
期刊:Science Translational Medicine
影响因子:14.6
doi:10.1126/scitranslmed.3001777
Passini, Marco A; Bu, Jie; Richards, Amy M; Kinnecom, Cathrine; Sardi, S Pablo; Stanek, Lisa M; Hua, Yimin; Rigo, Frank; Matson, John; Hung, Gene; Kaye, Edward M; Shihabuddin, Lamya S; Krainer, Adrian R; Bennett, C Frank; Cheng, Seng H
