A Chinese team has developed a rhesus monkey model of Duchenne muscular dystrophy, enabling it to evaluate a new gene therapy, with encouraging results.
- The DMDEx50 animal model has mutations in exon 50 of the DMD gene.
- A single-vector gene therapy called MyoAAV/Cas12iMax/sgRNA3Ex51 targeting exon 51 of the DMD gene was developed using Cas12iMax technology (similar to CRISPR/Cas9) and the MyoAAV vector targeting muscle.
- This therapy was developed so that it could be applied to both monkeys and humans.
- It was injected into a 17-month-old DMDEx50 monkey.
- One and a half years after administration, dystrophin expression was restored and muscle and motor functions improved.
- No safety problems were reported.
These positive results in a single animal provide proof of concept for this gene therapy based on gene editing, which has significant potential for clinical application. Further studies on a larger number of monkeys are essential before it can be evaluated in humans.
