Powis, Rachel A.Karyka, EvangeliaBoyd, PenelopeCome, JulienJones, Ross A.Zheng, YinanSzunyogova, EvaGroen, Ewout J. N.Hunter, GillianThomson, DerekWishart, Thomas M.Becker, Catherina G.Parson, Simon H.Martinat, CecileAzzouz, MimounGillingwater, Thomas H.2016-07-222016-07-222016-07-21Powis, R A, Karyka, E, Boyd, P, Come, J, Jones, R A, Zheng, Y, Szunyogova, E, Groen, E J N, Hunter, G, Thomson, D, Wishart, T M, Becker, C G, Parson, S H, Martinat, C, Azzouz, M & Gillingwater, T H 2016, 'Systemic restoration of UBA1 ameliorates disease in spinal muscular atrophy', JCI Insight, vol. 1, no. 11, e87908. https://doi.org/10.1172/jci.insight.879082379-3708ORCID: /0000-0002-8848-8738/work/82379608http://hdl.handle.net/2164/6245Acknowledgments Blood biochemistry analysis and serum analysis were performed by the Easter Bush Pathology Department, University of Edinburgh. Animal husbandry was performed by Centre for Integrative Physiology bio-research restructure technical staff, University of Edinburgh. Assistance with intravenous injections was provided by Ian Coldicott (University of Sheffield) and Hannah Shorrock (University of Edinburgh). Human blood cDNA was a gift to GH from Kathy Evans, University of Edinburgh. Imaging was performed at the IMPACT imaging facility, University of Edinburgh, with technical assistance from Anisha Kubasik-Thayil. The authors would also like to thank Lyndsay Murray for technical discussions relating to qRT-PCR analysis. This work was supported by funding from the SMA Trust and the Anatomical Society (via grants to THG); the Euan MacDonald Centre for Motor Neurone Disease Research (via grants to THG and SHP); the Wellcome Trust (via grants to EJNG and THG); Muscular Dystrophy UK (via grants to THG and CGB); a Elphinstone Scholarship from the University of Aberdeen (to SHP); and The French Muscular Dystrophy Association (via grants to CM and JC).173704212engR MedicineWellcome TrustSupplementary DataRJournal article10.1172/jci.insight.87908111