ScienceDaily (Feb. 27, 2009) β€” While scientists have identified one protein, dystrophin, as an important piece to curing muscular dystrophy, another part of the mystery has eluded scientists for the past 14 years. Now, scientists have identified the location of the genetic material responsible for a molecular compound that is vital to curing the disease…

Since 1994, researchers have known about the importance of nNOS, but have not been able to determine how to produce nNOS in a dystrophic muscle, or a muscle lacking dystrophin. Many scientists have tried to solve this mystery without success. In his most recent study, published Monday in The Journal of Clinical Investigation, Duan and his team identified the location of genetic material responsible for the production of nNOS.

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.”…

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