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Systemic restoration of UBA1 ameliorates disease in spinal muscular atrophy.
Powis RA, Karyka E, Boyd P, Côme J, Jones RA, Zheng Y, Szunyogova E, Groen EJ, Hunter G, Thomson D, Wishart TM, Becker CG, Parson SH, Martinat C, Azzouz M, Gillingwater TH. Powis RA, et al. Among authors: zheng y. JCI Insight. 2016 Jul 21;1(11):e87908. doi: 10.1172/jci.insight.87908. JCI Insight. 2016. PMID: 27699224 Free PMC article.
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