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...

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Publicado en:Mol Ther
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.
Formato: Artigo
Lenguaje:Inglês
Publicado: American Society of Gene & Cell Therapy 2020
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Original Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC6952030/
https://ncbi.nlm.nih.gov/pubmed/31628052
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2019.08.016
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