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2019 | 1 |
2022 | 2 |
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Page 1
In vivo delivery of CRISPR-Cas9 using lipid nanoparticles enables antithrombin gene editing for sustainable hemophilia A and B therapy.
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: kim ug.
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.
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Extru-seq: a method for predicting genome-wide Cas9 off-target sites with advantages of both cell-based and in vitro approaches.
Kwon J, Kim M, Hwang W, Jo A, Hwang GH, Jung M, Kim UG, Cui G, Kim H, Eom JH, Hur JK, Lee J, Kim Y, Kim JS, Bae S, Lee JK.
Kwon J, et al. Among authors: kim ug.
Genome Biol. 2023 Jan 10;24(1):4. doi: 10.1186/s13059-022-02842-4.
Genome Biol. 2023.
PMID: 36627653
Free PMC article.
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CRISPR-Cas9-mediated therapeutic editing of Rpe65 ameliorates the disease phenotypes in a mouse model of Leber congenital amaurosis.
Jo DH, Song DW, Cho CS, Kim UG, Lee KJ, Lee K, Park SW, Kim D, Kim JH, Kim JS, Kim S, Kim JH, Lee JM.
Jo DH, et al. Among authors: kim ug.
Sci Adv. 2019 Oct 30;5(10):eaax1210. doi: 10.1126/sciadv.aax1210. eCollection 2019 Oct.
Sci Adv. 2019.
PMID: 31692906
Free PMC article.
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