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AAV9-mediated SMN gene therapy rescues cardiac desmin but not lamin A/C and elastin dysregulation in Smn(2B/−) spinal muscular atrophy mice

Structural, functional and molecular cardiac defects have been reported in spinal muscular atrophy (SMA) patients and mouse models. Previous quantitative proteomics analyses demonstrated widespread molecular defects in the severe Taiwanese SMA mouse model. Whether such changes are conserved across d...

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Detalles Bibliográficos
Autores principales: Brown, Sharon J, Šoltić, Darija, Synowsky, Silvia A, Shirran, Sally L, Chilcott, Ellie, Shorrock, Hannah K, Gillingwater, Thomas H, Yáñez-Muñoz, Rafael J, Schneider, Bernard, Bowerman, Melissa, Fuller, Heidi R
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10549791/
https://www.ncbi.nlm.nih.gov/pubmed/37498175
http://dx.doi.org/10.1093/hmg/ddad121