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Chemical Biology and Therapeutics Science Program

Building the first base editors.

Waterbury AL, Iram I, Liau BB. Building the first base editors. Nature chemical biology. 2025;21(1):16-17. doi:10.1038/s41589-024-01790-3

In vivo base editing extends lifespan of a humanized mouse model of prion disease.

An M, Davis JR, Levy JM, et al. In vivo base editing extends lifespan of a humanized mouse model of prion disease. Nature medicine. 2025. doi:10.1038/s41591-024-03466-w

Directed evolution of engineered virus-like particles with improved production and transduction efficiencies.

Raguram A, An M, Chen PZ, Liu DR. Directed evolution of engineered virus-like particles with improved production and transduction efficiencies. Nature biotechnology. 2024. doi:10.1038/s41587-024-02467-x

In vivo photoreceptor base editing ameliorates rhodopsin-E150K autosomal-recessive retinitis pigmentosa in mice.

Du SW, Newby GA, Salom D, et al. In vivo photoreceptor base editing ameliorates rhodopsin-E150K autosomal-recessive retinitis pigmentosa in mice. Proceedings of the National Academy of Sciences of the United States of America. 2024;121(48):e2416827121. doi:10.1073/pnas.2416827121

EvoAI enables extreme compression and reconstruction of the protein sequence space.

Ma Z, Li W, Shen Y, et al. EvoAI enables extreme compression and reconstruction of the protein sequence space. Nature methods. 2024. doi:10.1038/s41592-024-02504-2

Introducing a G-Makassar variant in HSCs by in vivo base editing treats sickle cell disease in mice.

Li C, Georgakopoulou A, Paschoudi K, et al. Introducing a G-Makassar variant in HSCs by in vivo base editing treats sickle cell disease in mice. Molecular therapy : the journal of the American Society of Gene Therapy. 2024. doi:10.1016/j.ymthe.2024.10.018

Systematic optimization of prime editing for the efficient functional correction of CFTR F508del in human airway epithelial cells.

Sousa AA, Hemez C, Lei L, et al. Systematic optimization of prime editing for the efficient functional correction of CFTR F508del in human airway epithelial cells. Nature biomedical engineering. 2024. doi:10.1038/s41551-024-01233-3

Uncoupling histone modification crosstalk by engineering lysine demethylase LSD1.

Lee K, Barone M, Waterbury AL, et al. Uncoupling histone modification crosstalk by engineering lysine demethylase LSD1. Nature chemical biology. 2024. doi:10.1038/s41589-024-01671-9

Efficient site-specific integration of large genes in mammalian cells via continuously evolved recombinases and prime editing.

Pandey S, Gao XD, Krasnow NA, et al. Efficient site-specific integration of large genes in mammalian cells via continuously evolved recombinases and prime editing. Nature biomedical engineering. 2024. doi:10.1038/s41551-024-01227-1

Bone-marrow-homing lipid nanoparticles for genome editing in diseased and malignant haematopoietic stem cells.

Lian X, Chatterjee S, Sun Y, et al. Bone-marrow-homing lipid nanoparticles for genome editing in diseased and malignant haematopoietic stem cells. Nature nanotechnology. 2024. doi:10.1038/s41565-024-01680-8