Combining multiomics and drug perturbation profiles to identify muscle-specific treatments for spinal muscular atrophy

Spinal muscular atrophy (SMA) is a neuromuscular disorder caused by loss of survival motor neuron (SMN) protein. While SMN restoration therapies are beneficial, they are not a cure. We aimed to identify potentially novel treatments to alleviate muscle pathology combining transcriptomics, proteomics,...

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Detalles Bibliográficos
Autores principales: Meijboom, Katharina E., Volpato, Viola, Monzón-Sandoval, Jimena, Hoolachan, Joseph M., Hammond, Suzan M., Abendroth, Frank, de Jong, Olivier G., Hazell, Gareth, Ahlskog, Nina, Wood, Matthew J.A., Webber, Caleb, Bowerman, Melissa
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society for Clinical Investigation 2021
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8410072/
https://www.ncbi.nlm.nih.gov/pubmed/34236053
http://dx.doi.org/10.1172/jci.insight.149446

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8410072/
https://www.ncbi.nlm.nih.gov/pubmed/34236053
http://dx.doi.org/10.1172/jci.insight.149446

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