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Gene Therapy Yields Positive Results in Mice With Heart Condition

Update Date: Aug 20, 2014 05:43 PM EDT

Gene therapy protects mice from a life-threatening heart disease caused by muscular dystrophy, according to a new research. 

The gene therapy has been developed by researchers at the University of Missouri School of Medicine. 

"This is a new therapeutic avenue," said Yi Lai, Ph.D., the leading author of the study and assistant research professor in the MU School of Medicine's Department of Molecular Microbiology and Immunology, in the press release. "This is just a first step, but we hope this could lead to a treatment for people with this devastating heart condition, which is a leading cause of death for people with Duchenne muscular dystrophy."

About one in 3,500 children are born with Duchenne muscular dystrophy (DMD). The condition is responsible for progressive wasting away of muscles, starting in the legs and pelvis. As a result children affected with DMD face difficulty in walking and most need wheelchairs by age 12. 

 As the disorder depletes the skeletal muscles, it causes the heart to decay. As a result, weakened heart kills up to 40 percent of people with DMD. 

The study in question did not try to replace the faulty gene, instead it targets a different gene. The targeted gene is involved with the heart's built-in system for responding to heart attacks and other emergencies. 

"The study showed for the first time that a modified nNOS gene could be delivered through gene therapy to protect the hearts of mice from Duchenne muscular dystrophy," said Dongsheng Duan, Ph.D., co-author of the study and Margaret Proctor Mulligan Professor in Medical Research at the MU School of Medicine.

"Since nNOS protects against multiple heart diseases, this method could one day be extended to the treatment of other heart diseases, such as heart failure or a heart attack," Duan said.

The study has been published in the journal Human Molecular Genetics.

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