Title: | Systemic restoration of UBA1 ameliorates disease in spinal muscular atrophy |
Journal : | JCI insight, 1, 11 |
Authors: | Powis RA, Author ; Karyka E ; Boyd P ; Come J ; Jones RA ; Zheng Y ; Szunyogova E ; Groen EJ ; Hunter G ; Thomson D ; Wishart TM ; Becker CG ; Parson SH ; Martinat C ; Azzouz M ; Gillingwater TH |
Material Type: | Article |
Publication Date: | 2016 |
Languages: | English |
Keywords : | AAV9 vectors ; gene therapy ; spinal muscular atrophy ; spinal muscular atrophy mouse ; UBA1 gene ; UBA1 related disease ; ubiquitin-protein ligase |
Pubmed / DOI : | DOI : 10.1172/jci.insight.87908 / Pubmed : 27699224 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/27699224 |