University of Missouri scientist and his team have identified the location of the genetic material responsible for a molecular compound that is vital to curing the Muscular dystrophy (MD). The new advance will improve gene therapy strategies and they have turned muscle in mice with muscular dystrophy into regular muscle again.
Even if there is a cure for MD, it is likely that there will still be telethon on labor day for remaining medical issues. Even if Jerry Lewis stopped hosting the show and the disease was cured, it is likey there would be some labor day medical fundraiser.
Muscular dystrophy, which affects approximately 250,000 people in the United States, occurs when damaged muscle tissue is replaced with fibrous, bony or fatty tissue and loses function. While scientists have identified one protein, dystrophin, as an important piece to curing the disease, another part of the mystery has eluded scientists for the past 14 years.
Duchenne muscular dystrophy (DMD), predominantly affecting males, is the most common type of muscular dystrophy. Patients with Duchenne muscular dystrophy have a gene mutation that disrupts the production of dystrophin. Absence of dystrophin starts a chain reaction that eventually leads to muscle cell degeneration and death.
While dystrophin is vital for muscle development, the protein also needs several “helpers” to maintain the muscle tissue. One of these “helper” molecular compounds is nNOS, which produces nitric oxide. This is important for muscles that are in use during high intensity movements, such as exercise.
Following the identification of the genetic material, Duan and his team created a series of new dystrophin genes. In their study, they used dystrophic mice to test the efficacy of these new genes. After genetically correcting the mice with the new dystrophin gene, Duan’s team discovered that the missing nNOS was now restored in the dystrophic muscle. The mice that received the new gene did not experience muscle damage or fatigue following exercise.
“With this new discovery, we’ve solved a longstanding mystery of Duchenne Muscular Dystrophy,” Duan said. “This will change the way we approach gene therapy for DMD patients in the future. With this study, we have finally found the genetic material that can fully restore all the functions required for correcting a dystrophic muscle and turning it into a normal muscle.”
The MDA telethon