So far, there is no effective treatment for deteriorating muscle function in DMD patients. A promising approach for treating this life-threatening disease is gene transfer to restore dystrophin expression using a safe, non-pathogenic viral vector called adeno-associated viral (AAV) vector.
Can gene therapy cure muscular dystrophy?
How does gene therapy treat muscular dystrophy?
This gene therapy product leads to production of a shortened form of the dystrophin that addresses the underlying cause of DMD. This micro-dystrophin has the potential to halt the disease progression and could lead to physiological improvement for patients.”
When Will gene therapy be available for muscular dystrophy?
Pfizer plans to start its big clinical test by the end of the year. Solid, meanwhile, could dose more patients in its early stage study in the first quarter of 2021.
Is there a cure coming soon for muscular dystrophy?
There is currently no cure for the disease, and patients with DMD have an average life expectancy of just 26 years old. A mutation in the dystrophin gene, which is important for maintaining muscle fibers, causes DMD. Muscle fibers in people with DMD are highly susceptible to injury and are also unable to regenerate.
Can you fix muscular dystrophy?
Although there's no cure for any form of muscular dystrophy, treatment for some forms of the disease can help extend the time a person with the disease can remain mobile and help with heart and lung muscle strength. Trials of new therapies are ongoing.
Is gene therapy an option for DMD?
A gene therapy clinical trial may open up new possibilities for the treatment of Duchenne muscular dystrophy.
Does Crispr cure DMD?
CRISPR-Cas can be used to permanently repair the mutated DMD gene, leading to the expression of the encoded protein, dystrophin, in systems ranging from cells derived from DMD patients to animal models of DMD.
Can stem cells cure muscular dystrophy?
There are currently no stem-cell-based therapies for muscular dystrophy. Research has provided some exciting avenues for potentially effective future treatments. A lot of work is still needed to determine whether these treatments will be safe and effective in humans.
Can Muscular Dystrophy be reversed?
There's currently no cure for muscular dystrophy (MD), but a variety of treatments can help to manage the condition. As different types of MD can cause quite specific problems, the treatment you receive will be tailored to your needs.