After toying with the guide RNAs that are used to turn Cas9 into an effective gene editing tool, some of the scientific leaders in the field say they've been able to gain greater insights into how they can use the same approach to not only do surgery on DNA, but to also simultaneously control gene regulation, turning genes off and on with an eye to controlling a variety of diseases.
The team, combining scientists at the Wyss Institute at Harvard as well as MIT, found that shorter guide RNA no long cut into a gene but could control gene expression. By "toggling" the length of the guide RNA, they can employ it for either purpose with great accuracy. They've introduced a new kit that "allows the innate Cas9 protein from Streptococcus pyogenes to cleave certain genes while simultaneously regulating the expression of others through engineering the guide RNA," according to a statement. The researchers reported their findings in the September 7 issue of Nature Methods.
"By using our uncovered guide RNA principles, we can now for the first time toggle a single protein to gain direct control over both, gene sequences and gene expression, and turn almost any DNA sequence into a regulatory sequence to further bend the cell to our will," says George Church, one of the scientific leaders in the field of genetics, in a statement. "We envision future uses for the technology that can help decipher the tangled web of interactions underlying for example cancer drug resistance and stem cell differentiation, or design advanced synthetic gene circuitries."
"This new functionality will improve our ability to decipher the complex relationships between interdependent genes responsible for many diseases," said Marcelle Tuttle, a research fellow at the Wyss Institute and co-author of the study, in a statement.
Church is one of the academic founders of Editas, which appears to be in the lead among the first wave of biotechs pushing gene editing programs into the clinic. Over the last two years CRISPR/Cas9 technology has inspired a slate of biotech startups like Editas, Intellia and CRISPR Therapeutics, which are all hustling along preclinical efforts designed to apply the science to developing new medicines. In addition, academic groups around the world have been tinkering with CRISPR/Cas9 as well, tapping in on its cheap, accessible approach to treating a wide group of diseases.