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Systemic restoration of UBA1 ameliorates disease in spinal muscular atrophy

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dc.contributor.authorPowis, Rachel A.
dc.contributor.authorKaryka, Evangelia
dc.contributor.authorBoyd, Penelope
dc.contributor.authorCome, Julien
dc.contributor.authorJones, Ross A.
dc.contributor.authorZheng, Yinan
dc.contributor.authorSzunyogova, Eva
dc.contributor.authorGroen, Ewout J. N.
dc.contributor.authorHunter, Gillian
dc.contributor.authorThomson, Derek
dc.contributor.authorWishart, Thomas M.
dc.contributor.authorBecker, Catherina G.
dc.contributor.authorParson, Simon H.
dc.contributor.authorMartinat, Cecile
dc.contributor.authorAzzouz, Mimoun
dc.contributor.authorGillingwater, Thomas H.
dc.contributor.institutionUniversity of Aberdeen.Medical Sciencesen
dc.contributor.institutionUniversity of Aberdeen.Medical Educationen
dc.contributor.institutionUniversity of Aberdeen.The Suttie Centre - Anatomyen
dc.date.accessioned2016-07-22T13:55:03Z
dc.date.available2016-07-22T13:55:03Z
dc.date.issued2016-07-21
dc.descriptionAcknowledgments 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).en
dc.description.statusPeer revieweden
dc.format.extent17
dc.format.extent3704212
dc.identifier69013390
dc.identifiera2c604c8-23be-4cf3-9126-0b1bd07fe810
dc.identifier85055597996
dc.identifier.citationPowis, 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.87908en
dc.identifier.doi10.1172/jci.insight.87908
dc.identifier.iss11en
dc.identifier.issn2379-3708
dc.identifier.otherORCID: /0000-0002-8848-8738/work/82379608
dc.identifier.urihttp://hdl.handle.net/2164/6245
dc.identifier.vol1en
dc.language.isoeng
dc.relation.ispartofJCI Insighten
dc.subjectR Medicineen
dc.subjectWellcome Trusten
dc.subjectSupplementary Dataen
dc.subject.lccRen
dc.titleSystemic restoration of UBA1 ameliorates disease in spinal muscular atrophyen
dc.typeJournal articleen

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