Gene therapy may prevent heart transplants in kids

Children with genetic heart disease may one day no longer need medication, invasive procedures or transplants, thanks to a gene therapy breakthrough by Australian researchers.

Delivering a healthy copy of the ALPK3 gene in a single injection reversed heart muscle disease in lab-grown patient heart tissue and mouse models, a study led by researchers from the Murdoch Children’s Research Institute (MCRI) in Melbourne found.

 ALPK3 variants can cause cardiomyopathy, a group of diseases that affect the heart’s ability to pump blood, leading to enlarged hearts with weak, irregular heartbeats. The estimated 30 million people affected worldwide are at greater risk of heart failure and death, and treatment options are limited, researchers said.