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.
