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Get Free AccessBecker 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.
Marta Przymuszała, Alicja Martyniak, J Kwiatkowska, Jarosław Meyer-Szary, Karolina Śledzińska, Jolanta Wierzba, Jozef Dulak, Urszula Florczyk, Jacek Stępniewski (2024). Generation of human induced pluripotent stem cell line derived from Becker muscular dystrophy patient with CRISPR/Cas9-mediated correction of DMD gene mutation. , 76, DOI: https://doi.org/10.1016/j.scr.2024.103327.
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Type
Article
Year
2024
Authors
9
Datasets
0
Total Files
0
Language
en
DOI
https://doi.org/10.1016/j.scr.2024.103327
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