Funded MRC PhD at the University of Exeter using CRISPR epigenome editing and iPSC microglia to test which Alzheimer's epigenetic changes matter.

Sankaran and colleagues establish DNA methylation-based epigenome editing in human hematopoietic stem cells and their progeny. Targeting ITGB3, they show that silencing persists through self-renewal and megakaryocytic differentiation to alter platelet function and can be reversed through targeted demethylation. Extension to additional platelet genes demonstrates broad applicability.

Compare 19 single-cell method families across genome, epigenome, transcriptome, proteome, spatial, and multiomics, and learn how to choose the right one.

A handful of start-up firms are testing therapies that target specific epigenetic markers to treat everything from high cholesterol to a rare muscular disorder.

Original Article · Open Access

Study of almost 900 dogs from roughly 120 breeds finds that rapid ageing of large canines is linked to changes in the epigenome.

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...

Compare 19 single-cell method families across genome, epigenome, transcriptome, proteome, spatial, and multiomics, and learn how to choose the right one.

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...