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Chemical Biology and Therapeutics Science Program

Restoration of visual function in adult mice with an inherited retinal disease via adenine base editing.

Suh S, Choi EH, Leinonen H, et al. Restoration of visual function in adult mice with an inherited retinal disease via adenine base editing. Nature biomedical engineering. 2021;5(2):169-178. doi:10.1038/s41551-020-00632-6

Bifunctional modalities for repurposing protein function.

Modell AE, Lai S, Nguyen TM, Choudhary A. Bifunctional modalities for repurposing protein function. Cell chemical biology. 2021;28(7):1081-1089. doi:10.1016/j.chembiol.2021.06.005

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

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

Precision genome editing using cytosine and adenine base editors in mammalian cells.

Huang TP, Newby GA, Liu DR. Precision genome editing using cytosine and adenine base editors in mammalian cells. Nature protocols. 2021;16(2):1089-1128. doi:10.1038/s41596-020-00450-9

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

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

Laboratory evolution of a sortase enzyme that modifies amyloid-β protein.

Podracky CJ, An C, DeSousa A, Dorr BM, Walsh D, Liu DR. Laboratory evolution of a sortase enzyme that modifies amyloid-β protein. Nature chemical biology. 2021;17(3):317-325. doi:10.1038/s41589-020-00706-1

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

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