Today, Dr. Eric Olson spoke at the UT Southwestern Wellstone Muscular Dystrophy Center Duchenne Muscular Dystrophy Clinical Symposium. He presented promising pre-clinical data about gene editing using CRISPR/Cas9 for Duchenne. CureDuchenne is funding this research to advance Dr. Olson’s CRISPR gene editing development program that has the potential to correct 80% of Duchenne mutations.
Early data shows rescue of dystrophin following systemic delivery of AAV9-Cas9 in mice after four weeks. It also shows rescue of cardiac dystrophin following systemic delivery of AAV9-Cas9 of 80%. New data in dog models is showing an increase of more than 50% in expression of dystrophin at six weeks post single injection. There is no anticipation for any other animal models prior to human clinical trials.
Distinguishing features of gene editing include:
- Durable correction of the disease-causing mutation
- A one-time treatment, in principle
- Can potentially reach all affected cell types
- Can potentially correct up to 80% of Duchenne mutations
We are excited about the potential of Dr. Olson’s research but this is early science and there is much work to be done before entering clinical trials. Watch this video and view the infographic to learn more about CRISPR/Cas9.
CureDuchenne has committed $5M to accelerate Dr. Olson’s work. We are half way there. We need everyone’s help to make sure we can deliver those funds immediately to help advance this important work. Donate to help advance this research that could potentially help 80% of those with Duchenne.