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

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@article{Passini2011AntisenseOD, title={Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy.}, author={Marco A. Passini and Jie Bu and Amy M Richards and Cathrine Kinnecom and Sergio Pablo Sardi and Lisa M. Stanek and Yimin Hua and Frank Rigo and John E. Matson and Gene H Hung and Edward M. Kaye and Lamya S. Shihabuddin and Adrian R. Krainer and C. F. Bennett and Seng H. Cheng}, journal={Science translational medicine}, year={2011}, volume={3 72}, pages={72ra18} }