A ‘Cure for Heart Disease’? A Single Shot Succeeds in Monkeys – The New York Times

Not just did the system operate in 13 monkeys, the researchers reported, however it appeared that every liver cell was modified. After gene editing, the monkeys LDL levels stopped by 59 percent within two weeks. The ANGPTL3 gene editing led to a 64 percent decline in triglyceride levels.
One danger of gene editing is the procedure may lead to modification of DNA that researchers are not anticipating. “You will never have the ability to have no off-target results,” alerted Dr. Deepak Srivastava, president of the Gladstone Institutes in San Francisco.
In treating a condition as common as cardiovascular disease, he included, even an uncommon negative effects can suggest many clients are affected. Far, nevertheless, the scientists state that they have not seen any unintentional modifying of other genes.
Another question is how long the effect on cholesterol and triglyceride levels will last, Dr. Davidson stated. “We hope it will be one-and-done, but we have to validate that with scientific trials,” he said.
Jennifer Doudna, a biochemist of the University of California, Berkeley, and an innovator of Crispr, the revolutionary gene editing system, stated: “In concept, Verves method could be much better since its a one-time treatment.”
However it is much prematurely to state if it will be long-lasting and safe, she added.
If the strategy does work in humans, its greatest effect may be in poorer countries that can not manage costly injections for individuals at high threat of heart illness, said Dr. Daniel Rader, chairman of the department of genetics at the University of Pennsylvania and a member of Verves scientific board of advisers.
Dr. Kathiresan, of Verve, kept in mind that half of all first cardiac arrest end in unexpected death, making it important to safeguard those at high danger.