Aarhus Universitets segl

Ex vivo CRISPR repair and the creation of muscle stem cells for curing Duchenne muscular dystrophy

Projekter: ProjektForskning

Se relationer på Aarhus Universitet


This research project is funded by the Novo Nordisk Foundation.


Duchenne Muscular Dystrophy (DMD) is a progressive, severe, and
muscle-wasting disease affecting approximately 1 in 5000 live male
births. However, no effective drug is available for curing the disease.
Based on highly efficient CRISPR/Cas9 gene editing and stem cell
technologies, this project proposes to develop and consolidate a novel
therapy to cure DMD (EXOCURE). The EXOCURE is based on ex vivo
CRISPR/Cas9repair of DMD mutations in patients’ own skin cells. The D
MD-corrected cells will be converted into muscle stem cells by mRNA
reprogramming. These DMD-corrected muscle stem cells will be used in
the future as an off-the-shelf stem cell product to restore muscle
functions in DMD patients. The EXOCURE will create a personalized,
safe and effective therapy to cure DMD.
Effektiv start/slut dato01/01/202231/12/2024

ID: 228405141