Versatile And Robust Epigenetic Modulation Platform For Programmable Gene Activation And Repression

By Ryan Rickels, Vasu Kommireddy, Jasmine Bonanno, Karina Herlambang, Andy Chan, Megan Williamson, Isaiah Taylor, Melissa Porter, Hari Padmanabham, Allie Crawley, Amy Pooler, and Ron Chong

Epigenetic editing offers a way to regulate disease-implicated genes without changing the underlying DNA sequence. A modular, RNA-guided platform demonstrates potent and tunable gene activation and repression across metabolic, immunogenic, and neurodevelopmental targets. Results include durable B2M repression lasting at least four weeks after transient mRNA delivery, therapeutically relevant activation levels, and simultaneous activation and repression in the same human cells. Unbiased transcriptome profiling also indicates strong on-target specificity with minimal off-target activity.

With delivery options spanning nucleofection, lipid nanoparticles, and compact all-in-one AAV packaging, see how programmable epigenetic modulation could provide flexible control of gene expression across diverse cell types and therapeutic applications.