UC Berkeley researchers have found a way to improve revolutionary gene-editing technology CRISPR-Cas9 by restricting its activation to desired cells.
The study detailing the improved CRISPR-Cas 9, or ProCas9, was published Jan. 10 in the scientific journal Cell. CRISPR-Cas9, a bacterial system that cuts up invading viruses, has been repurposed as a gene-editing tool capable of cutting and pasting desired genetic sequences to modify the desired genome.
“This is an extra layer of security you could put on the molecule to ensure accurate cutting,” said campus associate professor of molecular and cell biology David Savage in a press release.
ProCas9 can be activated in specific cells through the use of a biological “turn-on” switch or an additional protein. The added protein must then be cut by a particular enzyme, including those found in cancer cells or dangerous pathogens, before ProCas9 can be activated, ensuring that ProCas9 is only used in desired cells.
“The key innovation here is both the method and accomplishment of creating a biomolecular lock around Cas9 such that only when delivered to the right cells, can the gene editor do its job,” said campus professor of bioengineering Adam Arkin in an email. “It can deliver the molecule anywhere but it will only be active where it is programmed to be active.”
ProCas9 has exhibited many potential medical applications, including its ability to fight viral pathogens in plants. According to the study text, ProCas9 can be activated by plant and human viruses.
Arkin said ProCas9 can also be used to modify or destroy targeted cells, making it an evolutionary innovation in the study of oncology.
“This could be delivered, for example, into cancer patients and only kill the cells that express specific cancer-associated proteases,” Arkin said in an email.