Monday, April 6, 2015

Gene therapy protects mice from lethal heart condition, researchers find


About 1 in 3,500 children, mostly boys are born, with Duchenne muscular dystrophy (DMD). They experience a progressive wasting away of muscles, starting in the legs and pelvis. Children with DMD have difficulty walking, and most need wheelchairs by age 12. As DMD depletes the skeletal muscles, it also causes the heart to decay. A weakened heart kills up to 40 percent of people with DMD, usually by their 20s or early 30s. DMD originates with mutations in a single gene. For more than two decades, researchers have explored using gene therapy, an experimental treatment, to replace the flawed gene with a healthy copy.
In a recent study conducted by the University of Missouri School of Medicine, researchers targeted a different gene that is involved with the heart's built-in system for responding to heart attacks and other emergencies rather than replacing the flawed gene like many others have attempted.
The gene that was targeted expresses a protein called nNOS, which activates briefly during short-term stresses to regulate the heart. Researchers altered the gene to enable more efficient transfer of the nNOS gene to mouse hearts. Seven months after the gene therapy, scientists found that the treatment has protected their hearts from the damage of DMD.
This technique is still in early stages and will need more researching before it is able to be applied on humans.
Although this technique is still in early development, I believe this technique has enormous potential not only in treating the damage that DMD causes on the heart, but the stress imposed by other diseases.

http://www.sciencenewsline.com/articles/2014082014360040.html
http://www.sciencedaily.com/releases/2014/08/140820110620.htm

1 comment:

  1. I think the findings of this study are impressive! I think it would be great if the damage that DMD causes on the heart could be treated. People affected by DMD would be able to live longer lives. Hopefully, it will not be long before trials using the technique can be performed on humans.

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