@article{article, title  = {{Systemic restoration of UBA1 ameliorates disease in spinal muscular atrophy}},

 publisher = {{The American Society for Clinical Investigation}},

 url = {{https://eprints.whiterose.ac.uk/id/eprint/102790 http://dx.doi.org/10.1172/jci.insight.87908 }},

 year = {{2016}},

 month = {{7}},

 author = {{Powis RA and Karyka E and Boyd P and Come J and Jones RA and Zheng Y and Szunyogova E and Groen EJN and Hunter G and Thomson D and Wishart TM et al}},

 doi = {{10.1172/jci.insight.87908}},

 volume = {{1}},

 journal = {{JCI Insight}},

 issue = {{11}},

 note = {{Accessed on 2025/11/24}}}