Gene editing figures to be part of the future of medicine, but a popular system for it has some room for improvement, researchers say.
That’s why a team from UC San Diego in La Jolla set out with Yale University researchers to develop a new system for gene editing that they believe may be safer and more efficient than the common current method known as CRISPR.
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CRISPR, short for “clustered regularly interspaced short palindromic repeats,” uses RNA and bacterial proteins to edit DNA. According to UCSD, it was adapted from a method used by bacteria as an immune defense against the DNA of viruses.
But when scientists use the method to edit human DNA, there can be unintended consequences such as genomic damage, said Aaron Smargon, first author of the study and an assistant project scientist in professor Gene Yeo’s lab at UCSD.
Several years ago, Smargon and Yeo got to work on a possible alternative that targeted small nuclear RNAs — RNA molecules that don’t make proteins but are located in the nucleus of cells — to swap out certain “letters” in the genetic code. Unlike CRISPR, this approach makes more specific, temporary modifications, researchers say.
The result is a tool that could have “wide applicability to a lot of complex genes that can accumulate a lot of mutations in the general population … that could be problematic,” Smargon said. “Beyond that, the small nuclear RNA we tested didn’t have off-target effects relative to state-of-the-art [technology], which suggests it could be a safer technology.”
He said the new tool also is capable of editing pre-mRNA, the precursor to the RNA that encodes protein genes, and long non-coding RNA, which has been “implicated in a number of diseases including neurodegenerative and cardiovascular,” he said. “So there are a lot of exciting applications” that could be personalized to treat individual diseases.
“We talk about precision medicine, and this is another arrow in the quiver that will, in some ways, make the treatments more effective and safer,” Smargon said. “In the scientific community, we are focused on developing therapeutics that are safer … that could really benefit patients the most.”
Though gene editing and CRISPR have been around for decades, it has been “amazing” to see how far the science has come, Smargon said.
“The CRISPR revolution has been and continues to be exhilarating,” he said. “Our work is adjacent to it because if CRISPR hadn’t happened, our work wouldn’t have happened. It’s been incredible to be part of the community and rapidly learn so much about genetics and learn from really great scientists and mentor new scientists. … We are seeing the first fruits of this labor.
“The benefits of CRISPR research will continue to compound. This century, you are going to see a lot of amazing new discoveries and technologies that will benefit society. The future is definitely bright.”