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.