CRISPR Can Now Hitch a Ride on Nanoparticles to Battle Disease
“CRISPR has a dirty secret: there’s really no perfect way to deliver the “molecular scissors” safely into cells. Most methods currently rely on viruses: the DNA that encodes the CRISPR machinery is spliced into a “viral vector” then injected into the troubled tissue.
That’s all well and good for diseases that affect blood and muscle. But for destinations buried deep within the body, delivery becomes a serious issue.
“We’re working on safer and more efficient delivery methods,” said the study’s lead author, Dr. Daniel Anderson, at the time.
This week his team finally delivered. They created a nanoparticle system that envelops the CRISPR components in a protective sphere. A skin prick sends the treatment on its way to the liver—no blasting or virus required.
In mice with genetically high cholesterol, the nanoparticles successfully turned off the faulty gene in over 80 percent of liver cells, bringing their blood lipid back to normal levels.
That’s the highest success rate ever achieved with CRISPR in adult animals…”
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