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Chemical Biology and Therapeutics Science Program

Chemical and optical control of CRISPR-associated nucleases.

Modell AE, Siriwardena SU, Shoba VM, Li X, Choudhary A. Chemical and optical control of CRISPR-associated nucleases. Current opinion in chemical biology. 2021;60:113-121. doi:10.1016/j.cbpa.2020.10.003

Disulfide-compatible phage-assisted continuous evolution in the periplasmic space.

Morrison MS, Wang T, Raguram A, Hemez C, Liu DR. Disulfide-compatible phage-assisted continuous evolution in the periplasmic space. Nature communications. 2021;12(1):5959. doi:10.1038/s41467-021-26279-8

Reconstruction of evolving gene variants and fitness from short sequencing reads.

Shen MW, Zhao KT, Liu DR. Reconstruction of evolving gene variants and fitness from short sequencing reads. Nature chemical biology. 2021;17(11):1188-1198. doi:10.1038/s41589-021-00876-6

In vivo somatic cell base editing and prime editing.

Newby GA, Liu DR. In vivo somatic cell base editing and prime editing. Molecular therapy : the journal of the American Society of Gene Therapy. 2021;29(11):3107-3124. doi:10.1016/j.ymthe.2021.09.002

Functional correction of CFTR mutations in human airway epithelial cells using adenine base editors.

Krishnamurthy S, Traore S, Cooney AL, et al. Functional correction of CFTR mutations in human airway epithelial cells using adenine base editors. Nucleic acids research. 2021;49(18):10558-10572. doi:10.1093/nar/gkab788

Efficient C•G-to-G•C base editors developed using CRISPRi screens, target-library analysis, and machine learning.

Koblan LW, Arbab M, Shen MW, et al. Efficient C•G-to-G•C base editors developed using CRISPRi screens, target-library analysis, and machine learning. Nature biotechnology. 2021;39(11):1414-1425. doi:10.1038/s41587-021-00938-z

Mechanisms of angiogenic incompetence in Hutchinson-Gilford progeria via downregulation of endothelial NOS.

Gete Y, Koblan LW, Mao X, et al. Mechanisms of angiogenic incompetence in Hutchinson-Gilford progeria via downregulation of endothelial NOS. Aging cell. 2021;20(7):e13388. doi:10.1111/acel.13388

Base editing of haematopoietic stem cells rescues sickle cell disease in mice.

Newby GA, Yen JS, Woodard K, et al. Base editing of haematopoietic stem cells rescues sickle cell disease in mice. Nature. 2021;595(7866):295-302. doi:10.1038/s41586-021-03609-w

Prime editing in mice reveals the essentiality of a single base in driving tissue-specific gene expression.

Gao P, Lyu Q, Ghanam A, et al. Prime editing in mice reveals the essentiality of a single base in driving tissue-specific gene expression. Genome biology. 2021;22(1):83. doi:10.1186/s13059-021-02304-3

Phage-assisted evolution of botulinum neurotoxin proteases with reprogrammed specificity.

Blum T, Liu H, Packer MS, et al. Phage-assisted evolution of botulinum neurotoxin proteases with reprogrammed specificity. Science (New York, N.Y.). 2021;371(6531):803-810. doi:10.1126/science.abf5972