Chemical Biology and Therapeutics Science Program

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 DOI PubMed

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 DOI PubMed

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 DOI PubMed

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 DOI PubMed

In vivo photoreceptor base editing ameliorates rhodopsin-E150K autosomal-recessive retinitis pigmentosa in mice.

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.2416827121Google Scholar DOI PubMed

High-throughput evaluation of genetic variants with prime editing sensor libraries.

Gould SI, Wuest AN, Dong K, et al. High-throughput evaluation of genetic variants with prime editing sensor libraries. Nature biotechnology. 2024. doi:10.1038/s41587-024-02172-9Google Scholar DOI PubMed

Phage-assisted evolution of highly active cytosine base editors with enhanced selectivity and minimal sequence context preference.

Zhang E, Neugebauer ME, Krasnow NA, Liu DR. Phage-assisted evolution of highly active cytosine base editors with enhanced selectivity and minimal sequence context preference. Nature communications. 2024;15(1):1697. doi:10.1038/s41467-024-45969-7Google Scholar DOI PubMed

Post-translational modification-centric base editor screens to assess phosphorylation site functionality in high throughput.

Kennedy PH, Sheikh AAD, Balakar M, et al. Post-translational modification-centric base editor screens to assess phosphorylation site functionality in high throughput. Nature methods. 2024. doi:10.1038/s41592-024-02256-zGoogle Scholar DOI PubMed

Efficient site-specific integration of large genes in mammalian cells via continuously evolved recombinases and prime editing.

Pandey S, Gao XD, Krasnow NA, et al. Efficient site-specific integration of large genes in mammalian cells via continuously evolved recombinases and prime editing. Nature biomedical engineering. 2024. doi:10.1038/s41551-024-01227-1Google Scholar DOI PubMed

Directed evolution of engineered virus-like particles with improved production and transduction efficiencies.

Raguram A, An M, Chen PZ, Liu DR. Directed evolution of engineered virus-like particles with improved production and transduction efficiencies. Nature biotechnology. 2024. doi:10.1038/s41587-024-02467-xGoogle Scholar DOI PubMed