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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Détails bibliographiques
Publié dans:Mol Ther
Auteurs principaux: 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.
Format: Artigo
Langue:Inglês
Publié: American Society of Gene & Cell Therapy 2020
Sujets:
Original Article
Accès en ligne: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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