Mifepristone alone and in combination with scAAV9-SMN1 gene therapy improves disease phenotypes in Smn 2B/- spinal muscular atrophy mice

Abstract Spinal muscular atrophy (SMA) is a neuromuscular disease caused by deletions or mutations in the survival motor neuron 1 (SMN1) gene. SMA is characterised by alpha motor neuron loss in the spinal cord and subsequent muscle atrophy. There are currently three approved SMN-directed therapies f...

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Principais autores:	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, Melissa Bowerman
Formato:	Artigo
Idioma:	Inglês
Publicado:	Nature Portfolio 2025-11-01
Series:	Scientific Reports
Assuntos:	Metabolism Klf15 Skeletal muscle Spinal muscular atrophy Mifepristone Combinatorial therapy
Acceso en liña:	https://doi.org/10.1038/s41598-025-24050-3
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