A one-off injection could one day lower your cholesterol levels for the rest of your life.
People born with natural mutations that disable a specific gene have a lower risk of heart disease, with no apparent side effects. Now a single injection has successfully disabled this same gene in animal tests for the first time.
This potential treatment would involve permanently altering the DNA inside some of the cells of a person’s body [with CRISPR], so doctors will have to be sure it is safe before trying it in people. But the benefits could be enormous. In theory, it could help millions live longer and healthier lives.
In 2005, it was discovered that a few people naturally have very low cholesterol levels, thanks to mutations that prevent their livers from making a protein called PCSK9…To mimic this effect, two companies have developed approved antibodies that remove the PCSK9 protein from the blood.
The big worry about using gene editing to alter DNA inside the body is that it could also cause unintended “off-target” mutations. In the worst case, these could turn cells cancerous.
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