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...

Täydet tiedot

Tallennettuna:
Bibliografiset tiedot
Julkaisussa:Mol Ther
Päätekijät: 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.
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: American Society of Gene & Cell Therapy 2020
Aiheet:
Original Article
Linkit: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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