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McCutcheon, S. R., Rohm, D., Iglesias, N. & Gersbach, C. A. Epigenome editing technologies for discovery and medicine. Nat. Biotechnol. 42, 1199–1217 (2024). Bendixen, L., Jensen, T. I. & Bak, R. O. CRISPR–Cas-mediated transcriptional modulation: the therapeutic promises of CRISPRa and CRISPRi. Mol. Ther. 31, 1920–1937 (2023). Matharu, N. et al. CRISPR-mediated activation of a promoter or enhancer rescues obesity caused by haploinsufficiency. Science 363, eaau0629 (2019). Liao, H. K. et al. In vivo target gene activation via CRISPR/Cas9-mediated trans-epigenetic modulation. Cell 171, 1495–1507 (2017). Dominguez, A. A., Lim, W. A. & Qi, L. S. Beyond editing: repurposing CRISPR–Cas9 for precision genome regulation and interrogation. Nat. Rev. Mol. Cell Biol. 17, 5–15 (2016). Tamura, S. et al. CRISPR activation for SCN2A-related neurodevelopmental disorders. Nature 646, 983–991 (2025). Tanenhaus, A. et al. Cell-selective adeno-associated virus-mediated SCN1A gene regulation therapy rescues mortality and seizure phenotypes in a Dravet syndrome mouse model and is well tolerated in nonhuman primates. Hum. Gene Ther. 33, 579–597 (2022). Clark, T. et al. CRISPR activation screens: navigating technologies and applications. Trends Biotechnol. 42, 1017–1034 (2024). Gilbert, L. A. et al. Genome-scale CRISPR-mediated control of gene repression and activation. Cell 159, 647–661 (2014). Konermann, S. et al. Genome-scale...

Human cells hold about two meters (6.6 feet) of DNA, folded and compacted to fit inside a nucleus. That packing is essential for life, but it creates a problem for scientists.

UCLA researchers map how mechanical forces open DNA, enabling more precise and efficient gene activation by CRISPR to boost cell conversion.

Gong et al. show that pancreatic tumors adopt a neural-like progenitor (NRP) program after cytotoxic injury and inflammation that resembles normal pancreas regeneration. Using CRISPRa-engineered organ...

Read the full paper: Combinatorial and Inducible CRISPRa/i Enables Canalized hiPSC Forward Programming and Iterative Refinement via Single-Cell Genomics

Five single-cell foundation models and two deep learning models were benchmarked on the standard CRISPRa doubles task. All of them had higher prediction error than a deliberately trivial additive baseline, and none beat predicting no change. The rebuttal says the metrics are miscalibrated. Either wa

Curtis and colleagues engineer a silencing-resistant, selectable CRISPRa platform in primary CAR T cells, enabling genome-wide survival-based screens that nominate endogenous genes to enhance CAR T cell fitness.

CRISPR activation (CRISPRa) enables precise up-regulation of gene expression for ex vivo and in vivo applications. However, a lack of scalable, high-coverage tools has limited comprehensive genetic sc...

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Read the full paper: A multiplexed gRNA piggyBac transposon system facilitates efficient induction of CRISPRi and CRISPRa in human pluripotent stem cells

Liu et al. demonstrate a cellular reprogramming strategy that directly transforms glioblastoma cells into functional dendritic cell-like cells. This in situ conversion remodels the tumor microenvironment and elicits robust, systemic antitumor immunity, offering a synergistic and promising approach for clinical cancer immunotherapy.