Gene therapy used to create 'biological pacemaker'

A study has revealed a minimally invasive gene transplant procedure that changes unspecialized heart cells into "biological pacemaker" cells that keep the heart steadily beating.

Eduardo Marban, the lead researcher, Cedars-Sinai Heart Institute, said that they have been able, for the first time, to create a biological pacemaker using minimally invasive methods and to show that the biological pacemaker supports the demands of daily life and that, they are also are the first to reprogram a heart cell in a living animal in order to effectively cure a disease.

He further explained that originally, they thought that biological pacemaker cells could be a temporary bridge therapy for patients who had an infection in the implanted pacemaker area, but, these results shows that with more research, they might be able to develop a long-lasting biological treatment for patients.

These laboratory findings could lead to clinical trials for humans who have heart rhythm disorders but who suffer side effects, such as infection of the leads that connect the device to the heart, from implanted mechanical pacemakers.

Eugenio Cingolani, MD, the director of the Heart Institute's Cardiogenetics-Familial Arrhythmia Clinic, said that in the future, pacemaker cells also could help infants born with congenital heart block.

According to Shlomo Melmed, dean of the Cedars-Sinai faculty, this work by Dr. Marban and his team heralds a new era of gene therapy, in which genes are used not only to correct a deficiency disorder, but to actually turn one kind of cell into another type.

This study is published in the print edition of the peer-reviewed journal Science Translational Medicine.