Systemic restoration of UBA1 ameliorates disease in spinal muscular atrophy

Rachel A. Powis, Evangelia Karyka, Penelope Boyd, Julien 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)

^*Corresponding author for this work