In a recent study published by Nature Biotechnology,1 Goudy et al.described an all-RNA CRISPRoff/CRISPRon platform that programs endogenous gene expression in primary human T-cells without introducing double-strand br...In a recent study published by Nature Biotechnology,1 Goudy et al.described an all-RNA CRISPRoff/CRISPRon platform that programs endogenous gene expression in primary human T-cells without introducing double-strand breaks,offering a solution to the longstanding safety limitations of nuclease-based multiplex editing.This platform demonstrates durable and locus-specific gene silencing or activation across multiple targets,improved in vivo tumor control,and a scalable,low-toxicity path for nextgeneration T-cell engineering.展开更多
基金A.E.O.acknowledges the NHMRC Clinical Trials and Cohort Studies Grant(APP2014763)and a fellowship from the NHMRC(1154650)This research was funded through a strategic research agreement with JDRF International(grant 2-SRA2024-1442-S-B)+1 种基金received additional support from a grant provided by the Danish Diabetes Academy to A.E.O.,which is financed by the Novo Nordisk Foundation(grant NNF17SA0031406)L.D.and Y.Y.acknowledge the China Scholarship Council(CSC 202506310014 and 202508130022,respectively).
文摘In a recent study published by Nature Biotechnology,1 Goudy et al.described an all-RNA CRISPRoff/CRISPRon platform that programs endogenous gene expression in primary human T-cells without introducing double-strand breaks,offering a solution to the longstanding safety limitations of nuclease-based multiplex editing.This platform demonstrates durable and locus-specific gene silencing or activation across multiple targets,improved in vivo tumor control,and a scalable,low-toxicity path for nextgeneration T-cell engineering.
