Advantages:
- Novel Therapeutic target to treat cardiac dystrophy in DMD: Nox4 Inhibition
- Potential to reduce fibrosis and improve cardiac function over conventional treatments
- Potential to combine NOX4 inhibition with dystrophin replacement therapies
Summary:
Duchenne Muscular Dystrophy (DMD) is a genetic disorder caused by mutations in the X-linked dystrophin gene that affects the structure and function of striated muscle (cardiac and skeletal muscle). Although DMD is primarily considered a skeletal muscle disorder due to its progressive muscle degeneration and weakness, patients with this condition also develop a gradual and progressive heart muscle disease, dystrophiccardiomyopathy.
Our researchers discovered a novel potential therapeutic target to reduce dystrophic cardiomyopathy, which has become a leading cause of fatalities in DMD patients. They identified CIP as a novel regulator of cardiomyopathy, which interacts with dystrophin to regulate dystrophic cardiomyopathy by mediating the NADPH oxidase 4 (Nox4) pathway. Their research demonstrates that Nox4 inhibitors have a positive effect on cardiac function and pathological tissue changes in two DMD mouse models.
In summary, Nox4 inhibition could be a novel cardiac-specific therapeutic approach for DMD. Since Nox4 inhibitors are already FDA approved for liver fibrosis, repurposing for DMD patients poses a promising and simple path through regulatory approval.
Nox4 inhibitor treatment rescues cardiac defects in mdx/CIP-/- mice: M-mode images for the control(right) and inhibitor(left) group of the 5m CIP KO/MDX mice. Quantitative EF%.
Desired Partnerships:
- License
- Sponsored Research
- Co-Development