A new version of the CRISPR-Cas9 gene-editing technology allows researchers to alter the genome not by slicing and dicing it, but by activating desirable genes. The method, which prompted improvements in mice with kidney disease, Type 1 diabetes, and muscular dystrophy, might be a useful complement to standard CRISPR-Cas9 methods.
“Some diseases can be treated using our system, whereas others will require gene correction via the traditional system,” say lead researchers Hsin-Kai Liao and Fumiyuki Hatanaka of the Salk Institute for Biological Studies in La Jolla, CA.
CRISPR-Cas9 is a gene-editing technique modified from the defense systems of bacteria. In traditional systems, a modified virus delivers a guide RNA (gRNA) and an enzyme called Cas9 into the target cell. The customizable gRNA directs the Cas9 to the stretch of DNA to be edited. The Cas9 snips the DNA and new segments of DNA can then be inserted as the cell repairs the broken genome.
CRISPR-Cas9 has shown great promise in animal studies, but using the technique in humans could be risky, Liao says, because slicing through double strands of DNA can create permanent mutations and increase the risk of cancer or other genetic problems.
The new technique avoids this risk. The team...
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