CRISPR gene editing has been at the forefront of the news these days – usually related to cancer research or hemophilia. However, an innovative study is taking this technology to the next level by using it to lower a person’s risk of a heart attack.
Today, millions of people take drugs like statins to lower their cardiovascular event risk. The use of statins doesn’t come without risks of its own, though. Add to that; they don’t fix the problem. They offer a temporary solution at best. Patients must continue to take the drug daily for it to be effective.
A new clinical trial by Verve Therapeutics investigates whether genome editing can offer a one-and-off solution that lowers an individual’s risk of having a heart attack.
What Is CRISPR Gene Editing?
The CRISPR gene editing technology mirrors a naturally occurring system bacteria use. By capturing small bits of DNA code from viruses, bacteria can create a process for identifying infection and producing RNA segments to disable the virus.
Researchers can take this same basic concept and adapt it to work in the human body. They develop a small piece of RNA that works as a guide to recognizing a place to cut a strand of DNA. Once cut, the scientists can add or delete sections of the genetic code. Verve is attempting to use this same system to lower the levels of LDL-C cholesterol that can increase the risk of a heart attack or stroke.
LDL-C cholesterol travels around the body in the extracellular fluid, transporting fat. The risk comes as the levels of this cholesterol rise. The body does have a way of combating rising cholesterol blood levels, but it doesn’t always work, especially as a person gets older.
Cells have a docking system that the LDL-C attaches to when necessary. Each cell controls how many of these docks it creates based on need. If the cells detect too much cholesterol in the blood, they develop docks, suck in the LDL-C and destroy it. That, in turn, removes excess junk in the bloodstream. The cell destroys these docks if they are not necessary.
It’s an imperfect system, though. The cells can inadvertently destroy too many docks, allowing the cholesterol to build up, where it can attach to the walls of blood vessels and restrict flow. That is how many heart attacks happen.
Is CRISPR Gene Therapy the Solution?
Researchers at biotechnology company Verve Therapeutics are looking to prove that there is a gene editing solution to lower the risk of a heart attack. Instead of trying to control blood cholesterol levels using statins, they hope to offer a one-time-only approach that involves gene editing to target the sources of the problem.
The team has had success in animal trials and is now moving on to human testing. This therapeutic approach edits the PCSK9 gene. PCSK9 is responsible for destroying the docks that bring cholesterol across cell membranes for elimination.
If it works, this is the first attempt at using based editors inside the body to create a solution that manages the risk of heart attacks. Time will tell if it is effective. The initial results should be available in 2023. If the process is successful, it could open the door for using this method for broader purposes.