Gene therapy acts as a protector against fatal heart condition

A dangerous heart condition associated with muscular dystrophy could one day be treated with a gene therapy that protects the heart under stress. Researchers at the University of Missouri (UM) are conducting a preclinical study to develop a gene therapy that could be used for treating other heart conditions, the institution announced last week.  

The UM researchers targeted a gene that expresses nNOS, a protein that goes into action when the heart is under elevated stress. This gene was altered to improve the transfer of nNOS genes in the hearts of mice.

“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,” said Dongsheng Duan, PhD, one of the researchers from the UM team, in the release.

Duchenne muscular dystrophy affects approximately one in 3,500 children, with a slant toward more boys than girls. The hallmark of this disease is muscle wasting, including the muscles of the heart, and up to 40 percent of those with the genetic disorder typically die of heart disease in their 20s or 30s.

The researchers recently published their findings in Human Molecular Genetics. Further research is needed before this technique could be used in a clinical setting.