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.