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Chemical Biology and Therapeutics Science Program

High-throughput evaluation of genetic variants with prime editing sensor libraries.

Gould SI, Wuest AN, Dong K, et al. High-throughput evaluation of genetic variants with prime editing sensor libraries. Nature biotechnology. 2024. doi:10.1038/s41587-024-02172-9

Phage-assisted evolution of highly active cytosine base editors with enhanced selectivity and minimal sequence context preference.

Zhang E, Neugebauer ME, Krasnow NA, Liu DR. Phage-assisted evolution of highly active cytosine base editors with enhanced selectivity and minimal sequence context preference. Nature communications. 2024;15(1):1697. doi:10.1038/s41467-024-45969-7

Twin prime editing mediated exon skipping/reinsertion for restored collagen VII expression in recessive dystrophic epidermolysis bullosa.

Steinbeck BJ, Gao XD, McElroy AN, et al. Twin prime editing mediated exon skipping/reinsertion for restored collagen VII expression in recessive dystrophic epidermolysis bullosa. The Journal of investigative dermatology. 2024. doi:10.1016/j.jid.2024.04.013

Systematic optimization of prime editing for the efficient functional correction of CFTR F508del in human airway epithelial cells.

Sousa AA, Hemez C, Lei L, et al. Systematic optimization of prime editing for the efficient functional correction of CFTR F508del in human airway epithelial cells. Nature biomedical engineering. 2024. doi:10.1038/s41551-024-01233-3

Engineered virus-like particles for transient delivery of prime editor ribonucleoprotein complexes in vivo.

An M, Raguram A, Du SW, et al. Engineered virus-like particles for transient delivery of prime editor ribonucleoprotein complexes in vivo. Nature biotechnology. 2024. doi:10.1038/s41587-023-02078-y

Efficient prime editing in two-cell mouse embryos using PEmbryo.

Kim-Yip RP, McNulty R, Joyce B, et al. Efficient prime editing in two-cell mouse embryos using PEmbryo. Nature biotechnology. 2024. doi:10.1038/s41587-023-02106-x

Post-translational modification-centric base editor screens to assess phosphorylation site functionality in high throughput.

Kennedy PH, Sheikh AAD, Balakar M, et al. Post-translational modification-centric base editor screens to assess phosphorylation site functionality in high throughput. Nature methods. 2024. doi:10.1038/s41592-024-02256-z

Efficient site-specific integration of large genes in mammalian cells via continuously evolved recombinases and prime editing.

Pandey S, Gao XD, Krasnow NA, et al. Efficient site-specific integration of large genes in mammalian cells via continuously evolved recombinases and prime editing. Nature biomedical engineering. 2024. doi:10.1038/s41551-024-01227-1

In vivo photoreceptor base editing ameliorates rhodopsin-E150K autosomal-recessive retinitis pigmentosa in mice.

Du SW, Newby GA, Salom D, et al. In vivo photoreceptor base editing ameliorates rhodopsin-E150K autosomal-recessive retinitis pigmentosa in mice. Proceedings of the National Academy of Sciences of the United States of America. 2024;121(48):e2416827121. doi:10.1073/pnas.2416827121

Directed evolution of engineered virus-like particles with improved production and transduction efficiencies.

Raguram A, An M, Chen PZ, Liu DR. Directed evolution of engineered virus-like particles with improved production and transduction efficiencies. Nature biotechnology. 2024. doi:10.1038/s41587-024-02467-x