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The SINE Compound KPT-350 Blocks Dystrophic Pathologies in DMD Zebrafish and Mice
Duchenne muscular dystrophy (DMD) is an X-linked muscle wasting disease that is caused by the loss of functional dystrophin protein in cardiac and skeletal muscles. DMD patient muscles become weakened, leading to eventual myofiber breakdown and replacement with fibrotic and adipose tissues. Inflamma...
Autores principales: | Hightower, Rylie M., Reid, Andrea L., Gibbs, Devin E., Wang, Yimin, Widrick, Jeffrey J., Kunkel, Louis M., Kastenschmidt, Jenna M., Villalta, S. Armando, van Groen, Thomas, Chang, Hua, Gornisiewicz, Savanna, Landesman, Yosef, Tamir, Sharon, Alexander, Matthew S. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Society of Gene & Cell Therapy
2020
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6952030/ https://www.ncbi.nlm.nih.gov/pubmed/31628052 http://dx.doi.org/10.1016/j.ymthe.2019.08.016 |
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