Funder
Number of items: 1.
Mifepristone alone and in combination with scAAV9- SMN1 gene therapy improves disease phenotypes in Smn 2B/- spinal muscular atrophy mice. (2025)
Emma R. Sutton,
Eve McCallion,
Joseph M. Hoolachan,
Özge Çetin,
Paloma Pacheco-Torres,
Saman Rashid,
Sihame Bouhmidi,
Katie Haynes,
Lauren Churchill,
Taylor Scaife,
Helena Chaytow,
Yu-Ting Huang,
Stephanie Duguez,
Bernard L. Schneider,
Thomas H. Gillingwater,
Maria Dimitriadi
and
Melissa Bowerman
