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CRISPR-Cas3 gene editing system restores dystrophin function in stem cells derived from patients with Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is a muscle degeneration disorder caused by mutations affecting the dystrophin gene. Researchers show how a dual CRISPR RNA method restored dystrophin protein function in induced pluripotent stem cells derived from DMD patients. The approach worked by removing large sections of the dystrophin gene, allowing the cells to skip faulty or misaligned sections of the genetic code. This yields truncated but still functional proteins for a wide variety of mutation patterns associated with DMD.

from Latest Science News -- ScienceDaily https://ift.tt/YC7ksFn

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