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Dual SMN inducing therapies can rescue survival and motor unit function in symptomatic ∆7SMA mice.
Neurobiol Dis. 2021 Nov;159:105488. doi: 10.1016/j.nbd.2021.105488. Epub 2021 Aug 20.
Neurobiol Dis. 2021.
PMID: 34425216
Free PMC article.
Intragenic complementation of amino and carboxy terminal SMN missense mutations can rescue Smn null mice.
McGovern VL, Kray KM, Arnold WD, Duque SI, Iyer CC, Massoni-Laporte A, Workman E, Patel A, Battle DJ, Burghes AHM.
McGovern VL, et al. Among authors: kray km.
Hum Mol Genet. 2020 Nov 1;29(21):3493-3503. doi: 10.1093/hmg/ddaa235.
Hum Mol Genet. 2020.
PMID: 33084884
Free PMC article.
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Base editing rescue of spinal muscular atrophy in cells and in mice.
Arbab M, Matuszek Z, Kray KM, Du A, Newby GA, Blatnik AJ, Raguram A, Richter MF, Zhao KT, Levy JM, Shen MW, Arnold WD, Wang D, Xie J, Gao G, Burghes AHM, Liu DR.
Arbab M, et al. Among authors: kray km.
Science. 2023 Apr 21;380(6642):eadg6518. doi: 10.1126/science.adg6518. Epub 2023 Apr 14.
Science. 2023.
PMID: 36996170
Free PMC article.
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