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Gene therapy created ‘biological pacemakers’
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Researchers have created a minimally invasive gene transplant procedure that turns unspecialized heart cells into “biological pacemaker” cells.
If the process proves safe and effective in humans, it could one day supplement or replace implanted mechanical pacemakers and/or help babies born with congenital heart block, the researchers said in a press release.
Eduardo Marbán, MD, PhD, director of the Cedars-Sinai Heart Institute, and colleagues previously observed that re-expression of the TBX18 gene would convert ordinary ventricular cardiomyocytes into induced sinoatrial node cells, also called pacemaker cells.
Eduardo Marbán
In the present study, the researchers injected seven pigs with TBX18 and five with green fluorescent protein, used as a control substance, to determine whether biological pacemaker activity could be created.
All seven pigs receiving TBX18 had faster heartbeats than the controls, and the discrepancy persisted throughout the 14 days of the study, the researchers found.
“Originally, we thought that biological pacemaker cells could be a temporary bridge therapy for patients who had an infection in the implanted pacemaker area,” Marbán stated in the release. “These results show us that with more research, we might be able to develop a long-lasting biological treatment for patients.”
Marbán added that the study is the first “to show that the biological pacemaker supports the demands of daily life. We are also the first to reprogram a heart cell in a living animal in order to effectively cure a disease.”
Another researcher, Eugenio Cingolani, MD, director of Cedars-Sinai Heart Institute’s Cardiogenics-Familial Arrhythmia Clinic, said the cells could eventually be of great use to infants with heart block.
“Babies still in the womb cannot have a pacemaker, but we hope to work with fetal medicine specialists to create a lifesaving catheter-based treatment for infants diagnosed with congenital heart block,” Cingolani said in the release. “It’s possible that one day, we may be able to save lives by replacing hardware with an injection of genes.”
If the next research steps are successful, human clinical trials could be in in about 3 years, Marbán said in the release.
Disclosure: The study was funded by the Cedars-Sinai Board of Governors and Cedars-Sinai Medical Center. Cedars-Sinai Medical Center has a patent pending on the use of TBX18 as a biological pacemaker. The researchers report no other relevant financial disclosures.
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