Pharmacology of a Central Nervous System Delivered 2′-O-Methoxyethyl–Modified Survival of Motor Neuron Splicing Oligonucleotide in Mice and Nonhuman Primates

作者：Frank Rigo, Seung Joo Chun, Daniel A. Norris, Gene Hung, Sam S. Lee, John E. Matson, Robert A. Fey, Hans J. Gaus, Yimin Hua, John S. Grundy, Adrian R. Krainer, Scott P. Henry, C. Frank Bennett · 发表于：Journal of Pharmacology and Experimental Therapeutics · 年份：2014 · DOI：10.1124/jpet.113.212407 · 被引用次数：283 · 研究领域：Neurogenetic and Muscular Disorders Research、RNA modifications and cancer、RNA Research and Splicing