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Pathological impact of SMN2 mis-splicing in adult SMA mice.
Sahashi K, Ling KK, Hua Y, Wilkinson JE, Nomakuchi T, Rigo F, Hung G, Xu D, Jiang YP, Lin RZ, Ko CP, Bennett CF, Krainer AR. Sahashi K, et al. Among authors: bennett cf. EMBO Mol Med. 2013 Oct;5(10):1586-601. doi: 10.1002/emmm.201302567. Epub 2013 Sep 9. EMBO Mol Med. 2013. PMID: 24014320 Free PMC article.
Pharmacology of a central nervous system delivered 2'-O-methoxyethyl-modified survival of motor neuron splicing oligonucleotide in mice and nonhuman primates.
Rigo F, Chun SJ, Norris DA, Hung G, Lee S, Matson J, Fey RA, Gaus H, Hua Y, Grundy JS, Krainer AR, Henry SP, Bennett CF. Rigo F, et al. Among authors: bennett cf. J Pharmacol Exp Ther. 2014 Jul;350(1):46-55. doi: 10.1124/jpet.113.212407. Epub 2014 Apr 30. J Pharmacol Exp Ther. 2014. PMID: 24784568 Free PMC article.
281 results