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Gene editing system restores dystrophin function in stem cells from patients with Duchenne muscular dystrophy
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Gene editing system restores dystrophin function in stem cells from patients with Duchenne muscular dystrophy

by Cell Press This photo shows green dystrophin protein restored by dual-Cas3 in skeletal muscle derived from DMD patient iPSCs. Credit: Akitsu HottaDuchenne muscular dystrophy (DMD) is a muscle degeneration disorder caused by mutations affecting the dystrophin gene. On August 24 in the journal Stem Cell Reports, researchers show how a dual CRISPR RNA method...