Project Grant 2227919

This Project Grant from the National Science Foundation's Technology, Innovation, and Partnerships program totaling $100,000 will support research to develop next-generation clustered regularly interspaced short palindromic repeat (CRISPR) systems for curing inherited diseases. Specifically, the awardee, the University of California, Berkeley, will work to circumvent in vivo delivery challenges with CRISPR-Cas9 by developing miniature RNA-guided genome editors smaller than Cas9 that can be easily packaged in viral vectors to access therapeutic targets not previously reachable. If successful, this research has the potential to revolutionize medicine by enabling the cure of genetic diseases currently only treatable through costly lifelong therapies. The compact genome editors proposed under this award, at approximately half the size of Cas9, aim to render previously untreatable genetic diseases accessible to gene editing therapies by facilitating in vivo delivery.