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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Bibliografski detalji
Izdano u:Mol Ther
Glavni autori: 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
Jezik:Inglês
Izdano: American Society of Gene & Cell Therapy 2020
Teme:
Original Article
Online pristup: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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