Generation of human induced pluripotent stem cell line derived from Becker muscular dystrophy patient with CRISPR/Cas9-mediated correction of DMD gene mutation


Becker muscular dystrophy (BMD) is an X-linked recessive disorder caused by in-frame deletions in the dystrophin gene (DMD), leading to progressive muscle degeneration and weakness. We generated a human induced pluripotent stem cell (hiPSC) line from a BMD patient. BMD hiPSCs were then engineered by CRISPR/Cas9-mediated knock-in of missing exons 3-9 of DMD gene. Obtained hiPSC line may be a valuable tool for investigating the mechanisms underlying BMD pathogenesis. Copyright © 2024 The Author(s). Published by Elsevier B.V. All rights reserved.

Authors Przymuszała M, Martyniak A, Kwiatkowska J, Meyer-Szary J, Śledzińska K, Wierzba J, Dulak J, Florczyk-Soluch U, Stępniewski J
Journal Stem cell research
Publication Date 2024 Apr;76:103327
PubMed 38324931
DOI 10.1016/j.scr.2024.103327

Research Projects

Cell Lines