Cardiac Myoediting Attenuates Cardiac Abnormalities in Human and Mouse Models of Duchenne Muscular Dystrophy

Conclusions: We show that CRISPR/Cas9-mediated correction of DMD ΔEx44 mitigates structural, functional and transcriptional abnormalities consistent with dilated cardiomyopathy irrespective of how the protein reading frame is restored. We show that these effects extend to postnatal editing in iPSC-CMs and mice. These findings provide key insights into the utility of genome editing as a novel therapeutic for DMD-associated cardiomyopathy.PMID:34372664 | DOI:10.1161/CIRCRESAHA.121.319579

Source: Circulation Research - August 10, 2021 Category: Cardiology Authors: Ayhan Atmanli Andreas C Chai Miao Cui Zhaoning Wang Takahiko Nishiyama Rhonda Bassel-Duby Eric N Olson Source Type: research