CRISPR Gene Editing Gets a Boost in Efficiency

Researchers from Duke Health found an enhanced approach to CRISPR that expands its functionality. The finding may lead to safer and more effective techniques for utilizing CRISPR technology as therapy.

“CRISPR is great, but there are a lot of places within the human genome that can't be edited well,” said senior author Bruce Sullenger, Ph.D. CRISPR depends on the guide RNA to direct it to the right position on DNA and enable the repair or deletion in that location. Frequently, the RNA molecule guide poses a challenge, since the editing or deletion process cannot proceed because the RNA molecule may not fold correctly or may be otherwise damaged. When that occurs, the guide RNA usually needs to be replaced with a new one, and the genome cannot be correctly targeted.

The team from Duke Health discovered a method to salvage a damaged guide RNA, which actually consists of two parts that must function harmoniously: an RNA sequence that detects the DNA target site, and a scaffolding sequence that keeps the enzyme in position to cut the DNA at the appropriate juncture. They discovered several RNA sequences that restored the scaffolding's integrity, thus proving that CRISPR gene editing is significantly more flexible than previously believed.

For more information, read the news release of Duke Health.