Mifepristone alone and in combination with scAAV9-SMN1 gene therapy improves disease phenotypes in Smn(2B/-) spinal muscular atrophy mice
米非司酮单独使用或与scAAV9-SMN1基因疗法联合使用，均可改善Smn(2B/-)脊髓性肌萎缩症小鼠的疾病表型。
期刊:Scientific Reports
影响因子:3.9
doi:10.1038/s41598-025-24050-3
Sutton, Emma R; McCallion, Eve; Hoolachan, Joseph M; Çetin, Özge; Pacheco-Torres, Paloma; Rashid, Saman; Bouhmidi, Sihame; Haynes, Katie; Churchill, Lauren; Scaife, Taylor; Chaytow, Helena; Huang, Yu-Ting; Duguez, Stephanie; Schneider, Bernard L; Gillingwater, Thomas H; Dimitriadi, Maria; Bowerman, Melissa
