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Base-editing corrects metabolic abnormalities in a humanized mouse model for glycogen storage disease type-Ia.
Arnaoutova I, Aratyn-Schaus Y, Zhang L, Packer MS, Chen HD, Lee C, Gautam S, Gregoire FM, Leboeuf D, Boule S, Fernandez TP, Huang V, Cheng LI, Lung G, Bannister B, Decker J, Leete T, Shuang LS, Bock C, Kothiyal P, Grayson P, Mok KW, Quinn JJ, Young L, Barrera L, Ciaramella G, Mansfield BC, Chou JY. Arnaoutova I, et al. Among authors: packer ms. Nat Commun. 2024 Nov 10;15(1):9729. doi: 10.1038/s41467-024-54108-1. Nat Commun. 2024. PMID: 39523369 Free PMC article.
Cytosine base editing inhibits hepatitis B virus replication and reduces HBsAg expression in vitro and in vivo.
Smekalova EM, Martinez MG, Combe E, Kumar A, Dejene S, Leboeuf D, Chen CY, Dorkin JR, Shuang LS, Kieft S, Young L, Barrera LA, Packer MS, Ciaramella G, Testoni B, Gregoire F, Zoulim F. Smekalova EM, et al. Among authors: packer ms. Mol Ther Nucleic Acids. 2023 Dec 27;35(1):102112. doi: 10.1016/j.omtn.2023.102112. eCollection 2024 Mar 12. Mol Ther Nucleic Acids. 2023. PMID: 38292874 Free PMC article.
Directed evolution of adenine base editors with increased activity and therapeutic application.
Gaudelli NM, Lam DK, Rees HA, Solá-Esteves NM, Barrera LA, Born DA, Edwards A, Gehrke JM, Lee SJ, Liquori AJ, Murray R, Packer MS, Rinaldi C, Slaymaker IM, Yen J, Young LE, Ciaramella G. Gaudelli NM, et al. Among authors: packer ms. Nat Biotechnol. 2020 Jul;38(7):892-900. doi: 10.1038/s41587-020-0491-6. Epub 2020 Apr 13. Nat Biotechnol. 2020. PMID: 32284586
18 results