Chemical Biology and Therapeutics Science Program

Proteolysis-targeting chimeras with reduced off-targets.

Nguyen TM, Sreekanth V, Deb A, et al. Proteolysis-targeting chimeras with reduced off-targets. Nature chemistry. 2023. doi:10.1038/s41557-023-01379-8Google Scholar PubMed DOI

Reciprocal mutations of lung-tropic AAV capsids lead to improved transduction properties.

Cooney AL, Brommel CM, Traore S, et al. Reciprocal mutations of lung-tropic AAV capsids lead to improved transduction properties. Frontiers in genome editing. 2023;5:1271813. doi:10.3389/fgeed.2023.1271813Google Scholar PubMed DOI

Shuttle peptide delivers base editor RNPs to rhesus monkey airway epithelial cells in vivo.

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-wGoogle Scholar PubMed DOI

Bifunctional Small Molecules That Induce Nuclear Localization and Targeted Transcriptional Regulation.

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.3c06179Google Scholar PubMed DOI

Base Editor Scanning Reveals Activating Mutations of DNMT3A.

Garcia EM, Lue NZ, Liang JK, et al. Base Editor Scanning Reveals Activating Mutations of DNMT3A. ACS chemical biology. 2023. doi:10.1021/acschembio.3c00257Google Scholar PubMed DOI

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/s41467-023-40476-7Google Scholar PubMed DOI

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.2023.06.020Google Scholar PubMed DOI

A general approach to identify cell-permeable and synthetic anti-CRISPR small molecules.

Lim D, Zhou Q, Cox KJ, et al. A general approach to identify cell-permeable and synthetic anti-CRISPR small molecules. Nat Cell Biol. 2022. doi:10.1038/s41556-022-01005-8Google Scholar PubMed DOI

Phenotypic Screening for Small Molecules that Protect β-Cells from Glucolipotoxicity.

Small JC, Joblin-Mills A, Carbone K, et al. Phenotypic Screening for Small Molecules that Protect β-Cells from Glucolipotoxicity. ACS Chem Biol. 2022. doi:10.1021/acschembio.2c00052Google Scholar PubMed DOI

Rational Design of Silicon-Based Zinc Ionophores.

Yamada K, Deb A, Shoba VM, et al. Rational Design of Silicon-Based Zinc Ionophores. Angew Chem Int Ed Engl. 2022:e202201698. doi:10.1002/anie.202201698Google Scholar PubMed DOI