Gene Therapy – Is HCM Cure Possible?

A team of international genetic researchers has just won “The Big Beat Challenge” – a grant from the British Heart Foundation of £30 million ($36 million) payable over a 5 years period to study potentially curative gene therapies to treat genetic cardiomyopathies.

The group, made up of leading scientists from the U.K., U.S. and Singapore, is called the Cure Heart Project and is led by Dr. Hugh Watkins, a cardiologist at the University of Oxford. The researchers plan to develop several different approaches aimed at specific mutations and develop them to the point where biotech companies will take over and conduct clinical trials.

According to Dr. Christine Seidman of Harvard University Medical School, the co-leader of the project, the goal is to edit the genetic code and restore the heart to normal function.  Recent progress has been made with respect to genetic therapy for other diseases, and the team is hopeful that this research will translate to genetic cardiomyopathies.

According to Dr. Watkins, the hope is to have a handful of therapies ready for clinical testing within 5 years.

Here are some articles about the grant describing the science involved:

The Guardian

Mirage News

Science

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