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Chemical Biology and Therapeutics Science Program

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

DrugMap: A quantitative pan-cancer analysis of cysteine ligandability.

Takahashi M, Chong HB, Zhang S, et al. DrugMap: A quantitative pan-cancer analysis of cysteine ligandability. Cell. 2024. doi:10.1016/j.cell.2024.03.027

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

EvoAI enables extreme compression and reconstruction of the protein sequence space.

Ma Z, Li W, Shen Y, et al. EvoAI enables extreme compression and reconstruction of the protein sequence space. Nature methods. 2024. doi:10.1038/s41592-024-02504-2

Introducing a G-Makassar variant in HSCs by in vivo base editing treats sickle cell disease in mice.

Li C, Georgakopoulou A, Paschoudi K, et al. Introducing a G-Makassar variant in HSCs by in vivo base editing treats sickle cell disease in mice. Molecular therapy : the journal of the American Society of Gene Therapy. 2024. doi:10.1016/j.ymthe.2024.10.018

Branched chemically modified poly(A) tails enhance the translation capacity of mRNA.

Chen H, Liu D, Guo J, et al. Branched chemically modified poly(A) tails enhance the translation capacity of mRNA. Nature biotechnology. 2024. doi:10.1038/s41587-024-02174-7

Bone-marrow-homing lipid nanoparticles for genome editing in diseased and malignant haematopoietic stem cells.

Lian X, Chatterjee S, Sun Y, et al. Bone-marrow-homing lipid nanoparticles for genome editing in diseased and malignant haematopoietic stem cells. Nature nanotechnology. 2024. doi:10.1038/s41565-024-01680-8

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

Continuous evolution of compact protein degradation tags regulated by selective molecular glues.

Mercer JAM, DeCarlo SJ, Burman SSR, et al. Continuous evolution of compact protein degradation tags regulated by selective molecular glues. Science (New York, N.Y.). 2024;383(6688):eadk4422. doi:10.1126/science.adk4422

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