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

Base editor scanning charts the DNMT3A activity landscape.

Lue NZ, Garcia EM, Ngan K, Lee C, Doench JG, Liau BB. Base editor scanning charts the DNMT3A activity landscape. Nature chemical biology. 2023;19(2):176-186. doi:10.1038/s41589-022-01167-4Google Scholar PubMed DOI

Photoswitchable Molecular Glues Enable Optical Control of Transcription Factor Degradation.

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:10.1101/2023.04.09.536172Google Scholar PubMed DOI

Photoswitchable Molecular Glues Enable Optical Control of Transcription Factor Degradation.

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:10.1101/2023.04.09.536172Google Scholar DOI

Assigning functionality to cysteines by base editing of cancer dependency genes.

Li H, Ma T, Remsberg JR, et al. Assigning functionality to cysteines by base editing of cancer dependency genes. Nature chemical biology. 2023. doi:10.1038/s41589-023-01428-wGoogle Scholar PubMed DOI

Nonviral base editing of KCNJ13 mutation preserves vision in a model of inherited retinal channelopathy.

Kabra M, Shahi PK, Wang Y, et al. Nonviral base editing of KCNJ13 mutation preserves vision in a model of inherited retinal channelopathy. The Journal of clinical investigation. 2023;133(19). doi:10.1172/JCI171356Google Scholar PubMed DOI

Ex vivo prime editing of patient haematopoietic stem cells rescues sickle-cell disease phenotypes after engraftment in mice.

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/s41551-023-01026-0Google Scholar PubMed DOI

Base editing rescue of spinal muscular atrophy in cells and in mice.

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.adg6518Google Scholar PubMed DOI

FALCON systematically interrogates free fatty acid biology and identifies a novel mediator of lipotoxicity.

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.2023.03.018Google Scholar PubMed DOI

In vivo HSC prime editing rescues sickle cell disease in a mouse model.

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.2022018252Google Scholar PubMed DOI