Dib C, Queenan J, Swartzrock L, et al. GFP-on mouse model for interrogation of in vivo gene editing. Nature communications. 2025;16(1):7017. doi:10.1038/s41467-025-61449-y
Chemical Biology and Therapeutics Science Program
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.2025.102486
Wu B, Chang H, Singh P, et al. Small molecule modulators of TOX protein re-invigorate T cell activity. bioRxiv : the preprint server for biology. 2025. doi:10.1101/2025.03.03.641115
Merz M, Shoba V, Pergu R, et al. Phosphorylation-inducing chimera rewires oncogenic kinase to trigger apoptosis. bioRxiv : the preprint server for biology. 2025. doi:10.1101/2025.06.13.659082
Witte IP, Lampe GD, Eitzinger S, et al. Programmable gene insertion in human cells with a laboratory-evolved CRISPR-associated transposase. Science (New York, N.Y.). 2025;388(6748):eadt5199. doi:10.1126/science.adt5199
Matuszek Ż, Arbab M, Kesavan M, et al. Base editing of trinucleotide repeats that cause Huntington’s disease and Friedreich’s ataxia reduces somatic repeat expansions in patient cells and in mice. Nature genetics. 2025. doi:10.1038/s41588-025-02172-8
Pergu R, Vedagopuram S, Kokkonda P, et al. Phosphorylation-inducing molecules for regulating dynamic cellular processes. bioRxiv : the preprint server for biology. 2025. doi:10.1101/2025.05.10.652895
Musunuru K, Grandinette S, Wang X, et al. Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease. The New England journal of medicine. 2025. doi:10.1056/NEJMoa2504747
Vedagopuram S, Sindi S, Chaudhary S, et al. Ultrasmall chemogenetic tags with group-transfer ligands. bioRxiv : the preprint server for biology. 2025. doi:10.1101/2025.05.10.653252
Eweje F, Ibrahim V, Shajii A, et al. Self-assembling protein nanoparticles for cytosolic delivery of nucleic acids and proteins. Nature biotechnology. 2025. doi:10.1038/s41587-025-02664-2