Chemical Biology and Therapeutics Science Program

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.2416827121Google Scholar PubMed DOI

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/s41592-024-02504-2Google Scholar PubMed DOI

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.2024.10.018Google Scholar PubMed DOI

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/s41551-024-01227-1Google Scholar PubMed DOI

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-xGoogle Scholar PubMed DOI

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.2024.04.013Google Scholar PubMed DOI

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.adk4422Google Scholar PubMed DOI

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-xGoogle Scholar PubMed DOI

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/s41565-024-01680-8Google Scholar PubMed DOI

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/s41551-024-01233-3Google Scholar PubMed DOI