A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells

Bhaskar Chanda Stem Cell Young et al. demonstrate restoration of the DMD reading frame by CRISPR/Cas9-mediated deletion of up to 725 kb in hiPSCs as a therapeutic strategy for 60% of Duchenne muscular dystrophy patients. The resulting internally deleted protein is shown to be functional in vitro and in vivo.

from Cell Stem Cell http://ift.tt/1PoFN0h
via Bhaskar Chanda UHN

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