Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy

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

Science translational medicine, 2011-03, Vol.3 (72), p.72ra18-72ra18 [Periódico revisado por pares]

United States: The American Association for the Advancement of Science

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