Single gene injection reverses inherited heart disease in mice and patient-grown tissue
Melbourne researchers have made a gene therapy breakthrough that could restore heart function in children with genetic heart disease, sparing them the need for transplants.
Melbourne researchers have developed a groundbreaking gene therapy that could potentially restore heart function in children with genetic heart disease, eliminating the need for transplants. The study, published in Nature Cardiovascular Research, demonstrates that a single injection of a healthy copy of the ALPK3 gene reversed heart muscle disease in both lab-grown patient heart tissue and mouse models.
ALPK3 variants can cause cardiomyopathy, a condition affecting about 30 million people worldwide, increasing the risk of heart failure and death. The therapy also showed promise in correcting other genetic heart diseases, such as those caused by the MYH7 and TTN truncating variants, which are the most common causes of dilated cardiomyopathy.
MCRI Dr. James McNamara emphasized that this discovery marks a significant step towards a cure for genetic heart disease. If successful in humans, this gene therapy could provide targeted treatment for a range of inherited heart diseases, sparing children from invasive procedures and long-term medication.
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