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In vivo delivery of CRISPR-Cas9 using lipid nanoparticles enables antithrombin gene editing for sustainable hemophilia A and B therapy.
Han JP, Kim M, Choi BS, Lee JH, Lee GS, Jeong M, Lee Y, Kim EA, Oh HK, Go N, Lee H, Lee KJ, Kim UG, Lee JY, Kim S, Chang J, Lee H, Song DW, Yeom SC. Han JP, et al. Among authors: lee y, lee h, lee gs, lee kj, lee jy, lee jh. Sci Adv. 2022 Jan 21;8(3):eabj6901. doi: 10.1126/sciadv.abj6901. Epub 2022 Jan 21. Sci Adv. 2022. PMID: 35061543 Free PMC article.
Genome editing-mediated knock-in of therapeutic genes ameliorates the disease phenotype in a model of hemophilia.
Lee JH, Oh HK, Choi BS, Lee HH, Lee KJ, Kim UG, Lee J, Lee H, Lee GS, Ahn SJ, Han JP, Kim S, Yeom SC, Song DW. Lee JH, et al. Among authors: lee h, lee j, lee gs, lee kj, lee hh. Mol Ther Nucleic Acids. 2022 Aug 4;29:551-562. doi: 10.1016/j.omtn.2022.08.002. eCollection 2022 Sep 13. Mol Ther Nucleic Acids. 2022. PMID: 36090746 Free PMC article.
961 results