Chemical Biology and Therapeutics Science Program

In vivo base editing of Angptl3 via lipid nanoparticles to treat cardiovascular disease.

Khirallah J, Bloomer H, Wich D, et al. In vivo base editing of Angptl3 via lipid nanoparticles to treat cardiovascular disease. Molecular therapy. Nucleic acids. 2025;36(2):102486. doi:10.1016/j.omtn.2025.102486Google Scholar PubMed DOI

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/s41589-024-01790-3Google Scholar PubMed DOI

Converging mechanism of UM171 and KBTBD4 neomorphic cancer mutations.

Xie X, Zhang O, Yeo MJR, et al. Converging mechanism of UM171 and KBTBD4 neomorphic cancer mutations. Nature. 2025. doi:10.1038/s41586-024-08533-3Google Scholar PubMed DOI

CTCF/RAD21 organize the ground state of chromatin-nuclear speckle association.

Yu R, Roseman S, Siegenfeld AP, et al. CTCF/RAD21 organize the ground state of chromatin-nuclear speckle association. Nature structural & molecular biology. 2025. doi:10.1038/s41594-024-01465-6Google Scholar PubMed DOI

Covalent adduct Grob fragmentation underlies LSD1 demethylase-specific inhibitor mechanism of action and resistance.

Waterbury AL, Caroli J, Zhang O, et al. Covalent adduct Grob fragmentation underlies LSD1 demethylase-specific inhibitor mechanism of action and resistance. Nature communications. 2025;16(1):3156. doi:10.1038/s41467-025-57477-3Google Scholar PubMed DOI

Systematic empirical evaluation of individual base editing targets: validating therapeutic targets in USH2A and comparison of methods.

Tachida Y, Manian K V, Butcher R, et al. Systematic empirical evaluation of individual base editing targets: validating therapeutic targets in USH2A and comparison of methods. Molecular therapy : the journal of the American Society of Gene Therapy. 2025. doi:10.1016/j.ymthe.2025.01.042Google Scholar PubMed DOI

A method for the detection and enrichment of endogenous cereblon substrates.

Lloyd HC, Li Y, Payne C, et al. A method for the detection and enrichment of endogenous cereblon substrates. bioRxiv : the preprint server for biology. 2025. doi:10.1101/2025.03.24.645063Google Scholar PubMed DOI

UM171 glues asymmetric CRL3-HDAC1/2 assembly to degrade CoREST corepressors.

Yeo MJR, Zhang O, Xie X, et al. UM171 glues asymmetric CRL3-HDAC1/2 assembly to degrade CoREST corepressors. Nature. 2025. doi:10.1038/s41586-024-08532-4Google Scholar PubMed DOI

Oxidation of retromer complex controls mitochondrial translation.

Zhang J, Ali MY, Chong HB, et al. Oxidation of retromer complex controls mitochondrial translation. Nature. 2025. doi:10.1038/s41586-025-08756-yGoogle Scholar PubMed DOI

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-wGoogle Scholar PubMed DOI