Published today in Nature Biotechnology, a new paper from IGI postdoc David Colognori, IGI bioinformatician Marena Trinidad, and IGI Founder Jennifer Doudna develops the CRISPR-CSM system for use in human and other eukaryotic cells. CRISPR-CSM is a type III CRISPR system, which targets RNA for cutting and degradation. Unlike other RNA-targeting technologies, CRISPR-CSM can reach RNA both in and outside of the nucleus, including the mRNAs that are used to make proteins and RNAs with other kinds of roles in the cell.
RNA-targeting technologies can not only be used to advance genomic research, but are actively being developed for use as human therapies in conditions like Huntington’s Disease, where a genetic variant causes cells to make toxic proteins that accumulate in clumps, disrupting cell function, or for combatting RNA viruses like influenza, Hepatitis C virus, Ebola virus, or coronaviruses. Right now, Doudna and her team are working on methods for delivering CRISPR-CSM to human cells for future therapies.
Read more: Colognori, D., Trinidad, M. & Doudna, J.A. Precise transcript targeting by CRISPR-Csm complexes. Nat Biotechnol (2023). https://doi.org/10.1038/s41587-022-01649-9