Young mice with hereditary heart muscle disease, known as hypertrophic cardiomyopathy (HCM), can be cured by means of gene therapy.
The team of researchers led by Prof. Dr. Lucie Carrier from the University Medical Center Hamburg Eppendorf (UKE) chose for their experiments mice with neonatal HCM. This severe form of HCM is caused by a common mutation of the gene encoding cardiac myosin-binding protein C (cMyBP-C). With the help of a harmless viral envelope - a transport vehicle commonly used in molecular biology - the investigators introduced the correct genetic material into the cells of the heart muscle. The diseased, one-day-old mice received a single dose of the therapeutic gene. In the period up to 34 weeks after treatment the researchers examined the morphology and pumping characteristics of the mice hearts. Hardly any differences were detected compared to healthy hearts. Moreover, the investigators found that the cardiomyocytes now produced roughly two thirds of the healthy cMyBP-C protein compared to only 10 percent prior to treatment.