Systemic restoration of UBA1 ameliorates disease in spinal muscular atrophy

Rachael A. Powis, Evangelina Karyka, Penelope Boyd, Julian Come, Ross A. Jones, Yinan Zheng, Eva Szunyogova, Ewout J.N. Groen, Gillian Hunter, Derek Thomson, Thomas M. Wishart, Catherina G. Becker, Simon H. Parson, Cecile Martinat, Mimoun Azzouz, Thomas H. Gillingwater*

*Corresponding author for this work

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