Article
Generation of human induced pluripotent stem cell line derived from Becker muscular dystrophy patient with CRISPR/Cas9-mediated correction of DMD gene mutation.
Stem cell research - 1 Apr 2024
Przymuszała Marta, Martyniak Alicja, Kwiatkowska Joanna, Meyer-Szary Jarosław, Śledzińska Karolina, Wierzba Jolanta, Dulak Józef, Florczyk-Soluch Urszula, Stępniewski Jacek
Abstract excerpt
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...
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