Compact CRISPR Tool Effectively Targets RNA

Salk Institute scientists have developed a newly identified RNA-targeting CRISPR-Cas system into a compact, easily delivered CRISPR -based tool for RNA knockdown in human cells. The team, led by Patrick Hsu, M.D., a Helmsley-Salk Fellow and senior author of the team’s published paper in Cell , describe use of the system, which they’ve dubbed CasRx, to correct protein imbalance in cells from a patient with frontotemporal dementia (FTD). “Our results present CasRx as a programmab