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Mifepristone alone and in combination with scAAV9-SMN1 gene therapy improves disease phenotypes in Smn2B/- spinal muscular atrophy mice.

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Title: Mifepristone alone and in combination with scAAV9-SMN1 gene therapy improves disease phenotypes in Smn2B/- spinal muscular atrophy mice.
Authors: Sutton, Emma R.1 (AUTHOR), McCallion, Eve1 (AUTHOR), Hoolachan, Joseph M.1 (AUTHOR), Çetin, Özge1 (AUTHOR), Pacheco-Torres, Paloma2 (AUTHOR), Rashid, Saman2 (AUTHOR), Bouhmidi, Sihame1 (AUTHOR), Haynes, Katie1 (AUTHOR), Churchill, Lauren3 (AUTHOR), Scaife, Taylor3 (AUTHOR), Chaytow, Helena4,5 (AUTHOR), Huang, Yu-Ting4,5 (AUTHOR), Duguez, Stephanie6 (AUTHOR), Schneider, Bernard L.7 (AUTHOR), Gillingwater, Thomas H.4,5 (AUTHOR), Dimitriadi, Maria2 (AUTHOR), Bowerman, Melissa1,8 (AUTHOR) m.bowerman@keele.ac.uk
Source: Scientific Reports. 11/17/2025, Vol. 15 Issue 1, p1-21. 21p.
Database: Academic Search Ultimate
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ISSN:20452322
DOI:10.1038/s41598-025-24050-3