Joynt AT, Kavanagh EW, Newby GA, et al. Protospacer modification improves base editing of a canonical splice site variant and recovery of CFTR function in human airway epithelial cells. Molecular therapy. Nucleic acids. 2023;33:335-350. doi:10.1016/j.omtn.2023.06.020
Chemical Biology and Therapeutics Science Program
Singh S, Tian W, Severance ZC, et al. Proximity-inducing modalities: the past, present, and future. Chemical Society reviews. 2023. doi:10.1039/d2cs00943a
Mayuranathan T, Newby GA, Feng R, et al. Potent and uniform fetal hemoglobin induction via base editing. Nature genetics. 2023. doi:10.1038/s41588-023-01434-7
Basile G, Vetere A, Hu J, et al. Excess pancreatic elastase alters acinar-β cell communication by impairing the mechano-signaling and the PAR2 pathways. Cell metabolism. 2023. doi:10.1016/j.cmet.2023.05.007
Peterson AA, Liu DR. Small-molecule discovery through DNA-encoded libraries. Nature reviews. Drug discovery. 2023. doi:10.1038/s41573-023-00713-6
Chen L, Hong M, Luan C, et al. Adenine transversion editors enable precise, efficient A•T-to-C•G base editing in mammalian cells and embryos. Nature biotechnology. 2023. doi:10.1038/s41587-023-01821-9
Peters CW, Hanlon KS, Ivanchenko M V, et al. Rescue of hearing by adenine base editing in a humanized mouse model of Usher syndrome type 1F. Molecular therapy : the journal of the American Society of Gene Therapy. 2023. doi:10.1016/j.ymthe.2023.06.007
Kwok HS, Freedy AM, Siegenfeld AP, et al. Drug addiction unveils a repressive methylation ceiling in EZH2-mutant lymphoma. Nature chemical biology. 2023. doi:10.1038/s41589-023-01299-1
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
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