Research

How can we engineer cells and biomolecules for improved therapeutics and to better understand human cell biology? This question has motivated our team to pioneer technologies for programming and studying biomedical functions in human cells since opening the lab in 2018. These technology development efforts build upon our expertise in genomics, synthetic biology, genome editing, and human gene regulation. In this research area, we primarily focus on designing genetic sequences for predictable gene expression and sense-and-respond functionality in live cells, creating synthetic “epi-editors” for precise control over cellular transcription and the human epigenome, and developing cutting-edge vector and gene delivery systems with improved safety and programmability. We are committed to making our technologies robust and plug-and-play to ensure broad accessibility and immediate utility for biomedical research and the scientific community at large.

Nearly every human disease displays some component of disrupted gene expression. Additionally, nearly all cellular functions arise through dynamic engagement between transcription factors and/or chromatin modifiers and the human genome. Thus, fully understanding how human genes are expressed has the potential to broadly advance the therapeutic landscape and enable biotechnological applications where precisely engineered cellular functions are required. To realize this tremendous promise, our team develops sophisticated programmable synthetic transcription factors and chromatin modifiers that enable selective and tunable control over the timing, extent, and duration of human gene expression. These efforts help advance the understanding of, and ability to harness, cellular transcription – which in turn accelerates biomedical discovery and paves the way for more robust gene and cell-based therapeutics.

Our team uses cutting-edge cell engineering and synthetic biology tools to manipulate human cells for diverse translational applications. Our research in this rapidly expanding area includes engineering human immune cells for robust performance in immunotherapy, developing novel vector systems and genomic regulatory elements for precise control of therapeutic payloads, and enhancing the efficacy of epi-editors in primary human cells – including immune cells, neurons, and hematopoietic stem cells. We are also interested in targeted in vivo delivery platforms, developing sophisticated cell-based and in vivo models of human disease, and building therapeutic “cell factories” for both ex vivo and in vivo use. Together, these efforts aim to generate safer, more programmable cell and gene therapies to meet pressing unmet clinical needs.