Next-Generation SINE Compound KPT-8602 Ameliorates Dystrophic Pathology in Zebrafish and Mouse Models of DMD.

Autor: English KG; Department of Pediatrics, Division of Neurology at Children's of Alabama the University of Alabama at Birmingham, Birmingham, AL 35233, USA., Reid AL; Department of Pediatrics, Division of Neurology at Children's of Alabama the University of Alabama at Birmingham, Birmingham, AL 35233, USA., Samani A; Department of Pediatrics, Division of Neurology at Children's of Alabama the University of Alabama at Birmingham, Birmingham, AL 35233, USA., Coulis GJF; Department of Physiology and Biophysics, University of California-Irvine, Irvine, CA 92697, USA.; Institute for Immunology, University of California-Irvine, Irvine, CA 92967, USA., Villalta SA; Department of Physiology and Biophysics, University of California-Irvine, Irvine, CA 92697, USA.; Institute for Immunology, University of California-Irvine, Irvine, CA 92967, USA., Walker CJ; Karyopharm Therapeutics, Newton, MA 02459, USA., Tamir S; Karyopharm Therapeutics, Newton, MA 02459, USA., Alexander MS; Department of Pediatrics, Division of Neurology at Children's of Alabama the University of Alabama at Birmingham, Birmingham, AL 35233, USA.; UAB Center for Exercise Medicine (UCEM), Birmingham, AL 35205, USA.; Department of Genetics, University of Alabama at Birmingham, Birmingham, AL 35233, USA.; UAB Civitan International Research Center (CIRC), Birmingham, AL 35233, USA.; UAB Center for Neurodegeneration and Experimental Therapeutics (CNET), Birmingham, AL 35294, USA.

Publikováno v: Biomedicines [Biomedicines] 2022 Sep 26; Vol. 10 (10). Date of Electronic Publication: 2022 Sep 26.