The CRISPR genome-editing method of removing cells from the body, editing their DNA, and then replacing them is showing promise for treating a wide variety of diseases, ranging from liver disease to muscular dystrophy, according to a report in
The CRISPR approach requires a protein that can cut DNA and a piece of RNA that delivers it to the site, and the challenge has been the delivery. In one murine experiment, fatty particles successfully delivered the CRISPR components to the liver, reversing the genetic condition transthyretin amyloidosis, while in another mouse study gold nanoparticles were used to repair genes associated with muscular dystrophy. Human trials may soon be on the horizon.
But safety remains a concern, especially when viruses are used for delivery because they can persist in cells, increasing the risk for erroneous further cutting of the DNA.