Davis JR, Banskota S, Levy JM, et al. Efficient prime editing in mouse brain, liver and heart with dual AAVs. Nature biotechnology. 2023. doi:10.1038/s41587-023-01758-z
Chemical Biology and Therapeutics Science Program
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
Everette KA, Newby GA, Levine RM, et al. Ex vivo prime editing of patient haematopoietic stem cells rescues sickle-cell disease phenotypes after engraftment in mice. Nature biomedical engineering. 2023. doi:10.1038/s41551-023-01026-0
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
Wieder N, Fried J, Kim C, et al. FALCON systematically interrogates free fatty acid biology and identifies a novel mediator of lipotoxicity. Cell metabolism. 2023. doi:10.1016/j.cmet.2023.03.018
Li C, Georgakopoulou A, Newby GA, et al. In vivo HSC prime editing rescues sickle cell disease in a mouse model. Blood. 2023;141(17):2085-2099. doi:10.1182/blood.2022018252
Kwok HS, Freedy AM, Siegenfeld AP, et al. Drug addiction unveils a repressive methylation ceiling in EZH2-mutant lymphoma. Nature chemical biology. 2023. doi:10.1038/s41589-023-01299-1
Arbab M, Matuszek Z, Kray KM, et al. Base editing rescue of spinal muscular atrophy in cells and in mice. Science (New York, N.Y.). 2023:eadg6518. doi:10.1126/science.adg6518
Sreekanth V, Jan M, Zhao KT, et al. A molecular glue approach to control the half-life of CRISPR-based technologies. bioRxiv : the preprint server for biology. 2023. doi:10.1101/2023.03.12.531757
McAuley GE, Yiu G, Chang PC, et al. Human T cell generation is restored in CD3δ severe combined immunodeficiency through adenine base editing. Cell. 2023;186(7):1398-1416.e23. doi:10.1016/j.cell.2023.02.027