Health
Genetic heart disease can only be cured in the “decisive moment” | Heart disease
Scientists are set to develop the world’s first cure for genetic heart disease by rewriting DNA in a move that is welcomed as the “decisive moment” in cardiovascular medicine.
A global team of experts from the United Kingdom, the United States and Singapore are working together to design an arm jab for patients to save the lives of thousands after receiving a £ 30 million grant from the British Heart Foundation. increase.
The team will design and test the first treatment for hereditary cardiomyopathy using a precision gene technique called base and prime editing for the first time in the heart with the goal of silencing defective genes. Animal testing has already shown that the technique works.
“This is a crucial moment in cardiovascular medicine,” said Professor Sir Nilesh Samani, medical director at BHF.
Hereditary myocardial conditions are caused by a variety of abnormalities in the heart that can cause sudden death and progressive heart failure. About 260,000 people in the UK are in this state and can die suddenly at any age.
Every week in the UK, 12 people under the age of 35 die of undiagnosed heart disease. This is often due to hereditary cardiomyopathy, also known as hereditary cardiomyopathy.
Everyone with hereditary cardiomyopathy has a 50/50 risk of inheriting the defective gene in each child, and often several members of the same family develop heart failure or require a heart transplant. , Die at a young age.
The team behind the new study was selected by an advisory board chaired by Professor Sir Patrick Valence, Principal Scientific Advisor to the UK Government.
Professor Hugh Watkins of Oxford University CureHeart projectSaid that cardiomyopathy is “really common” and affects 1 in 250 people worldwide.
“This is a once-in-a-generation opportunity to free our families from sudden death, heart failure, and the constant worry of the potential need for a heart transplant,” he said. “Thirty years of research have uncovered many genes that cause a variety of cardiomyopathy, specific genetic defects, and how they work. Over the next five years, clinical trials of gene therapy I believe I’m ready to start with. “
Under the new research program, experts hope to permanently correct or silence the mutated genes involved in causing these heart problems.
Christine Seedman, a professor of medicine at Harvard Medical School in the United States and co-leader of the CureHeart project, said the idea was to “fix the mind” and bring it back to normal functioning.
“Most of the mutations we see in human patients have a huge number of mutations, [mutations] – They all change one letter of the DNA code frequently, “she said. “This made it more likely that we could change that one letter to restore the code and create a normal gene with normal functioning.”
She said some “very elegant chemistry” has already advanced the field of science, adding:
“We may be able to offer these treatments prior to the disease. Individuals known by genetic testing are at an extraordinary risk of developing the disease and progressing to heart failure. It has never been possible to provide and that is the purpose of our project. We know it can be done and aim to get started. “
Sources 2/ https://www.theguardian.com/society/2022/jul/29/genetic-heart-conditions-could-be-cured-first-time-defining-moment The mention sources can contact us to remove/changing this article |
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