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Chemical Biology and Therapeutics Science Program

Rescue of hearing by adenine base editing in a humanized mouse model of Usher syndrome type 1F.

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

Adenine transversion editors enable precise, efficient A•T-to-C•G base editing in mammalian cells and embryos.

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

Small-molecule discovery through DNA-encoded libraries.

Peterson AA, Liu DR. Small-molecule discovery through DNA-encoded libraries. Nature reviews. Drug discovery. 2023. doi:10.1038/s41573-023-00713-6

Excess pancreatic elastase alters acinar-β cell communication by impairing the mechano-signaling and the PAR2 pathways.

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

Potent and uniform fetal hemoglobin induction via base editing.

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

Proximity-inducing modalities: the past, present, and future.

Singh S, Tian W, Severance ZC, et al. Proximity-inducing modalities: the past, present, and future. Chemical Society reviews. 2023. doi:10.1039/d2cs00943a

Protospacer modification improves base editing of a canonical splice site variant and recovery of CFTR function in human airway epithelial cells.

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

Treatment of monogenic and digenic dominant genetic hearing loss by CRISPR-Cas9 ribonucleoprotein delivery in vivo.

Tao Y, Lamas V, Du W, et al. Treatment of monogenic and digenic dominant genetic hearing loss by CRISPR-Cas9 ribonucleoprotein delivery in vivo. Nature communications. 2023;14(1):4928. doi:10.1038/s41467-023-40476-7

Photoswitchable Molecular Glues Enable Optical Control of Transcription Factor Degradation.

Arp CJ, Reynders M, Sreekanth V, et al. Photoswitchable Molecular Glues Enable Optical Control of Transcription Factor Degradation. bioRxiv : the preprint server for biology. 2023. doi:10.1101/2023.04.09.536172

Photoswitchable Molecular Glues Enable Optical Control of Transcription Factor Degradation.

Arp CJ, Reynders M, Sreekanth V, et al. Photoswitchable Molecular Glues Enable Optical Control of Transcription Factor Degradation. bioRxiv : the preprint server for biology. 2023. doi:10.1101/2023.04.09.536172