Functionally repaired DMD heart cells with Crispr-Cas9

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A team of American researchers has treated lines of induced pluripotent stem cells taken from a patient with Duchenne muscular dystrophy (DMD) with a deletion of exon 44 of the DMD gene with Crispr-Cas9. The aim was to restore the reading frame or to skip exon 45.

From the two iPS cell lines obtained, they derived cardiomyocytes in which they were able to: 

  • obtain a truncated but functional dystrophin; 
  • restore the morphology and structure of heart cells;
  • reduce the arrhythmia of heart cells. In a model of mice with DMD, injection of this Crispr-Cas9 product into neonates restored dystrophin expression and reduced cardiac abnormalities in adult mice (18-22 months).

These results confirm the feasibility of this technique on heart cells affected by DMD. 

 

Cardiac Myoediting Attenuates Cardiac Abnormalities in Human and Mouse Models of Duchenne Muscular Dystrophy. Atmanli A, Chai AC, Cui M, Wang Z, Nishiyama T, Bassel-Duby R, Olson EN. Circ Res. 2021 Sep 3;129(6):602-616.

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