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Chemical Biology and Therapeutics Science Program
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
Lue NZ, Garcia EM, Ngan K, Lee C, Doench JG, Liau BB. Base editor scanning charts the DNMT3A activity landscape. Nature chemical biology. 2023;19(2):176-186. doi:10.1038/s41589-022-01167-4
Ely ZA, Mathey-Andrews N, Naranjo S, et al. A prime editor mouse to model a broad spectrum of somatic mutations in vivo. Nature biotechnology. 2023. doi:10.1038/s41587-023-01783-y
Kulhankova K, Traore S, Cheng X, et al. Shuttle peptide delivers base editor RNPs to rhesus monkey airway epithelial cells in vivo. Nature communications. 2023;14(1):8051. doi:10.1038/s41467-023-43904-w
Gibson WJ, Sadagopan A, Shoba VM, Choudhary A, Meyerson M, Schreiber SL. Bifunctional Small Molecules That Induce Nuclear Localization and Targeted Transcriptional Regulation. Journal of the American Chemical Society. 2023;145(48):26028-26037. doi:10.1021/jacs.3c06179
Ngan KCH, Hoenig SM, Kwok HS, et al. Activity-based CRISPR scanning uncovers allostery in DNA methylation maintenance machinery. eLife. 2023;12. doi:10.7554/eLife.80640
Li C, Georgakopoulou A, Newby GA, et al. In vivo HSC prime editing rescues sickle cell disease in a mouse model. Blood. 2023;141(17):2085-2099. doi:10.1182/blood.2022018252
Wieder N, Fried J, Kim C, et al. FALCON systematically interrogates free fatty acid biology and identifies a novel mediator of lipotoxicity. Cell metabolism. 2023. doi:10.1016/j.cmet.2023.03.018
Arbab M, Matuszek Z, Kray KM, et al. Base editing rescue of spinal muscular atrophy in cells and in mice. Science (New York, N.Y.). 2023:eadg6518. doi:10.1126/science.adg6518