A group of RNA experts say they've developed a potential upgrade for the wildly popular CRISPR/Cas9 gene editing tech that has been picked up in labs around the world.
Researchers drawn from Isis Pharmaceuticals ($ISIS), UC San Diego and the Ludwig Institute have created specially modified RNA--"designer" RNA--that can be used to guide Cas9, the cutting tool that is used to scissor into DNA, either inactivating a target gene or beginning the surgical repair of genes that cause disease. Their work was published in the Proceedings of the National Academy of Sciences.
Currently, a guide RNA is used to direct Cas9, but this new method is temporary. Editing stops when the designer RNA is cleared or another RNA drug can be added that brings the process to a halt.
"The RNA-based drugs we developed in this study provide many advantages over the current CRISPR/Cas9 system, such as increased editing efficiency and potential selectivity," said Don Cleveland, distinguished professor and chair of the department of cellular and molecular medicine at the UC San Diego School of Medicine and head of Ludwig Cancer Research's Laboratory for Cell Biology, in a release. "In addition, they can be synthesized efficiently, on an industrial scale and in a commercially feasible manner today."
Cleveland is a consultant to Isis, which has been developing RNA drugs for years. A group of prominent biotechs has been pioneering CRISPR's use in drug development. Editas got out in front, followed by CRISPR Therapeutics and Intellia, which is working with Novartis ($NVS).
"These findings provide a platform for multiple therapeutic applications, especially for nervous system diseases, using successive application of designer CRISPR RNA drugs," he added.