'Doctors in the US have announced plans for a radical gene therapy that aims to drastically reduce the risk of heart attack, the world’s leading cause of death, with a one-off injection.
The researchers hope to trial the therapy within the next three years in people with a rare genetic disorder that makes them prone to heart attacks in their 30s and 40s. If the treatment proves safe and effective in the patients, doctors will seek approval to offer the jab to a wider population.
“The therapy will be relevant, we think, to any adult at risk of a heart attack,” said Sekar Kathiresan, a cardiologist and geneticist at Harvard Medical School who will lead the effort. “We want this not only for people who have heart attacks at a young age because of a genetic disorder, but for garden variety heart attacks as well.”
Heart disease is the No 1 killer in many countries. An estimated 18m deaths are attributed to the condition every year, the vast majority of which, about 85%, are caused by heart attacks and strokes.
People who are at risk of a heart attack are typically put on a range of medicines, such as blood thinners, cholesterol-lowering statins, and pills for high blood pressure. Most must be taken daily for the rest of the person’s life, but many drift off their medication over time.
With a one-off gene therapy that permanently protects against heart attacks, the researchers hope to transform not only the impact heart disease has on lives, but the costs that health services face in caring for patients. Cardiovascular disease accounts for a quarter of all deaths in England and costs the NHS £7bn a year.
“The most exciting part of this is changing from a chronic care approach to a ‘one and done’ approach,” said Kathiresan. “We really think we can turn the tide against coronary disease by moving from a chronic care model to [eradication with] a one-time treatment.”'
Read more: One-off injection may drastically reduce heart attack risk
Genetic editing via CRISPR may cause widespread cancer, study warns
Any time a new technology has an industry buzzing, there will always be skeptics. When it comes to scientific advancements like genetic engineering and gene editing, much of that concern relates to the long-term effects of these seemingly miraculous practices. Even if something appears to be safe right now, how can anyone be certain it won’t cause problems five, 15, or 50 years down the line? Proponents used to laugh off these concerns, but a new game-changing study clearly illustrates the cancer-causing potential of genetic editing.
Some experts have said that CRISPR-Cas9, which entails altering specific genes in cells, could help us overcome diseases like cystic fibrosis, HIV, and Alzheimer’s. It almost sounded too good to be true, which should have been the first red flag. Now, the journal Nature Medicine has printed two studies that show the treatment could trigger cancer.
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One study was carried out by Novartis and involved pluripotent stem cells, while the other was carried out by Karolinska Institute and focused on retinal cells. Both of the studies in question focus on the p53 gene, which plays a role in preventing tumors by killing cells that have damaged DNA. Past research has shown that most tumors in humans cannot form when p53 is working correctly.
P53 also defends the body from the genome changes made in CRISPR-Cas9. When DNA is snipped and replaced using the procedure, p53 essentially causes the edited cells to self-destruct, which is why CRISPR fails so often. When CRISPR does work, however, it could be because the p53 in the cell doesn’t work as it should. Faulty p53 is also considered a precursor to several types of cancer.'
Read more: Genetic editing via CRISPR may cause widespread cancer, study warns