Could rewriting DNA cure genetic heart conditions?
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A team of experts from the United Kingdom, the United States, and Singapore will collaborate to create a jab that aims to save thousands of lives by rewriting DNA.
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Genetic heart conditions could be cured for the first time.
Scientists will rewrite DNA to provide the world's first remedy for genetic heart diseases, in what has been dubbed a "defining moment" in cardiovascular medicine.
Following a £30 million ($37 million) grant from the British Heart Foundation, a global team of experts from the UK, US, and Singapore is collaborating to design a shot in the arm for patients in order to save thousands of lives.
For the first time, the researchers will apply precision genetic techniques in the heart known as base and prime editing to build and test the first cure for inherited heart muscle illnesses, with the goal of silencing faulty genes. Animal experiments have already demonstrated that the procedures work.
“This is a defining moment for cardiovascular medicine,” said Professor Nilesh Samani, the BHF’s medical director.
Inherited heart muscle problems are caused by various cardiac defects and may result in sudden death or progressive heart failure. In the UK, approximately 260,000 people suffer from this life-threatening condition.
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Every week, 12 persons under the age of 35 die in the United Kingdom due to an undiagnosed cardiac illness, which is frequently caused by a hereditary heart muscle disease known as genetic cardiomyopathy.
All people with hereditary cardiomyopathies have a 50% chance of passing on defective genes to their children. Several members of the same family frequently develop heart disease, require a heart transplant, or die at an early age.
An advisory body chaired by Professor Patrick Vallance, the UK government's main scientific adviser, chose the team behind the new research.
“This is our once-in-generation opportunity to relieve families of the constant worry of sudden death, heart failure, and the potential need for a heart transplant,” he said. “After 30 years of research, we have discovered many genes and specific genetic faults responsible for different cardiomyopathies, and how they work. We believe we will have a gene therapy ready to start testing in clinical trials in the next five years.”
Experts believe the new research initiative will permanently fix or silence mutant genes causing these heart disorders.
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