Antisense Oligonucleotides Delivered to the Mouse CNS Ameliorate Symptoms of Severe Spinal Muscular Atrophy
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- 2 March 2011
- journal article
- research article
- Published by American Association for the Advancement of Science (AAAS) in Science Translational Medicine
- Vol. 3 (72), 72ra18
- https://doi.org/10.1126/scitranslmed.3001777
Abstract
Central nervous system–directed antisense therapy ameliorates symptoms in a severe neuromuscular disorder in mice.Keywords
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