MDA-supported researchers have used genetic engineering to target renegade cells of the immune system in mice with a disease resembling myasthenia gravis
posted on October 25, 2012 - 10:47am
Researchers funded in part by MDA say a gene-based therapy designed to treat myasthenia gravis (MG) has shown promise in mice with an MG-like disease.
The research team was led by Dan Drachman, a longtime MDA research grantee at Johns Hopkins University in Baltimore, who also co-directs the MDA neuromuscular disease clinic at that institution. Drachman, a professor of neurology, has a special...
Real progress is being made in supplying functional dystrophin genes to treat Duchenne muscular dystrophy (DMD), a disease in which mutated dystrophin genes keep this critical protein from being produced in muscle fibers.