Double-strand break-free epigenetic programming: a safer path for T-cell therapies
Abstract
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 long-standing 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 next-generation T-cell engineering.
Fields of Study & Subjects
CAR-T cell therapy research
Acute Lymphoblastic Leukemia research
Pluripotent Stem Cells Research
Publication details
- Journal / source
- Signal Transduction and Targeted Therapy
- Publisher
- Springer Science and Business Media LLC
- Volume
- 11
- Issue
- 1
- Pages
- 11–11
- Publication date
- 12 January 2026
- Language
- EN