A single injection disables the same gene responsible for increasing the risk for heart failure. Experts have successfully tested the drug in animals. Now the future is bright for people with high cholesterol and heart complications.
According to experts, the treatment will involve permanent altering of DNA inside some of the cells of a person’s body. This new drug could help millions of people live longer and healthier lives.
At a genomics meeting in London held this February, Lorenz Mayr, of pharmaceutical firm AstraZeneca, described the results of the animal tests. Though Mayr didn't confirm whether AstraZeneca plans to pursue this approach, he was excited to share the findings.
“The idea would be to do it as a one-off,” he later told New Scientist
. “It should be permanent.”
Researchers used the CRISPR technique to disable human versions of the PCSK9 gene in mice. PCSK9 genes are responsible for more cholesterol in the blood. People who lack these genes have more bad cholesterol absent in the blood.
The future is bright for people with high cholesterol with this new injectable drug (c) Shutterstock
Thus, researchers injected the CRISPR Cas 9 protein and a guiding RNA sequence into the animals. The RNA guide helps the Cas9 protein bind to a specific site in the gene. It then cuts the gene at that point, and when the break is repaired, errors that disable the gene are likely to be introduced.
Results showed that the mice given the CRISPR treatment had lower cholesterol levels than those which are injected with the antibody drugs.
However, the conundrum is that this gene editing technique to alter DNA inside the body may also cause unintended “off-target” mutations. In the worst case, these could turn cells cancerous.
According to Mayr, the team has tested for off-target effects in 26 different tissues in the mice, and that the results will be published soon. “It’s very promising in terms of safety,” he told New Scientist.
Experts are now developing modified versions of the CRISPR protein that are so precise off-target effects occur no more often than natural mutations in cells. However, Gilles Lambert at the University of Reunion Island, who studies PCSK9 says, the desired outcome is, “for now it’s very far-fetched.”