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Scientists seek to develop gene therapy for heart disease and eliminate the risk of sudden death

Given by injection in the arm, the therapy could stop the heart disorder in those who already have genetic cardiomyopathies.

The British Heart Foundation (BHF) has awarded more than $36 million to genetic researchers who launched a leading project to correct the specific genes responsible for genetic heart conditions such as sudden death in young people. The international research team CureHeart, which received the grant, says it is confident its therapy, which involves rewriting DNA to edit or silence defective genes, could be ready for testing in human clinical trials within five years, this report reported. Friday at a press release the BHF.

CureHeart is an initiative that brings together the best scientists specializing in inherited cardiomyopathy from the UK, the US and Singapore. The team has identified the specific genes and genetic errors that cause genetic heart conditions, as well as how they work. The group is a pioneer in the development of the first cures for different hereditary cardiomyopathies.

They have already been doing preliminary work in animals and human cells. Administered through an injection in the arm, the therapy could stop the heart disorder among those already living with genetic cardiomyopathies, they say. And it could prevent the development of the disease in family members who carry a defective gene but have not yet developed the condition, effectively curing the problem.

Professor Hugh Watkins, of the University of Oxford and principal investigator of the CureHeart project, said that cardiomyopathies are “really common” and affect one in 250 people in the world (about 260,000 in the UK). Every week in the UK, 12 people under the age of 35 die from an undiagnosed heart condition, most often caused by an inherited disease of the heart muscle.

All People with genetic cardiomyopathies have a 50-50 risk of passing defective genes to each of their children, and often multiple members of the same family develop heart failure, need a heart transplant, or die at a young age.

Although the symptoms of cardiomyopathy can usually be controlled with medications, devices such as pacemakers, or surgery, there is currently no cure for heart failure.

“This is our only chance in a generation to relieve families of constant worry about sudden death, heart failure and the possible need for a heart transplant,” said Watkins.

Professor Sir Nilesh Samani, Medical Director of the Fund British Heart Association, said: “This is a defining moment for cardiovascular medicine. CureHeart could not only be the creator of the first cure for inherited heart muscle diseases by tackling killer genes found in family trees, it could also usher in a new era in precision cardiology.”

He then added that “once successful, the same gene-editing innovations could be used to treat a wide range of common heart conditions in which genetic errors play a role.” . This would have a transformative impact and offer hope to thousands of families around the world affected by these devastating diseases.”


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