Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy - Passini, MA; Bu, J; Richards, AM; Kinnecom, C; Sardi, SP; Stanek, LM; Hua, Y; Rigo, F; Matson, J; Hung, G; Kaye, EM; Shihabuddin, LS; Krainer, AR; Bennett, CF; Cheng, SH (Sci Transl Med, 2011)