Can a type of gene therapy technology help people with heart muscle disease?

Antisense oligonucleotide-mediated correction of inherited cardiomyopathy

Duchenne muscular dystrophy (DMD) is an inherited disease – where loss of a protein called dystrophin leads to progressive muscle wasting, including degeneration of heart muscle. People with this disease die earlier. New treatments are needed to help people with this condition live longer and have a better quality of life. Professor Matthew Wood’s laboratory at the University of Oxford focuses on developing new treatments for neurological and muscle diseases, such as Parkinson’s and Duchenne Muscular Dystrophy (DMD). DMD is caused by a faulty version of a protein called dystrophin, which has an important role in muscle cells. Professor Wood’s team have now developed a gene therapy technology using molecules called antisense oligonucleotides that correct the underlying genetic fault in DMD in the skeletal and heart muscle cells of mice, restoring a version of the missing dystrophin protein. This grant will allow the researchers to see if these molecules work in mice, and work out if it can prevent or reverse the heart muscle failure that occurs in Duchenne muscular dystrophy (DMD). This work may reveal a new way to prevent or delay the onset of heart problems in DMD and prolong the lives of people with this condition.