Gene Therapy: Injection to Replace Heart Pumps and Transplants
By Jake Stern
Scientists from Imperial College London are testing an injection that could, in the future, replace heart transplants and other cardiac surgery. The subjects of the study are two dozen patients at Harefield hospital in the United Kingdom with serious heart failure and fitted with mechanical pumps. The gene therapy involves injecting a benign virus with copies of an enzyme that helps heart muscles contract by recycling calcium.
The replacement gene, called Mydicar, is developed by the US biotech company Celladon and will be given to 16 individuals in the study.
The head of the British Heart Foundation’s centre of regenerative medicine at Imperial, Professor Sian Harding, asserts “We will be using state-of-the-art methods to gain detailed information on how and where the gene therapy takes effect, which will potentially help us develop and improve the therapy.” The researchers will also test to see if the therapy works in patients with antibodies to the wild vaccine delivering the gene.“ We have adapted the wild virus by removing the viral genes and replacing them with the treatment SERCA gene, so the virus acts like a biological courier to deliver our treatment gene,” says Dr Alex Lyon, lead investigator from the National Heart & Lung Institute at Imperial.
Heart disease is an illness where the heart becomes weaker as its cells malfunction and is usually caused by high blood pressure, alcohol abuse, smoking, genetic defects or infections. Currently, about three million people in the US and Britain suffer from some degree of heart failure.
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