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Sleeping Beauty mRNA-LNP enables stable rAAV transgene expression in mouse and NHP hepatocytes and improves vector potency.
Zakas PM, Cunningham SC, Doherty A, van Dijk EB, Ibraheim R, Yu S, Mekonnen BD, Lang B, English EJ, Sun G, Duncan MC, Benczkowski MS, Altshuler RC, Singh MJ, Kibbler ES, Tonga GY, Wang ZJ, Wang ZJ, Li G, An D, Rottman JB, Bhavsar Y, Purcell C, Jain R, Alberry R, Roquet N, Fu Y, Citorik RJ, Rubens JR, Holmes MC, Cotta-Ramusino C, Querbes W, Alexander IE, Salomon WE. Zakas PM, et al. Mol Ther. 2024 Oct 2;32(10):3356-3371. doi: 10.1016/j.ymthe.2024.06.021. Epub 2024 Jul 8. Mol Ther. 2024. PMID: 38981468
Molecular coevolution of coagulation factor VIII and von Willebrand factor.
Zakas PM, Coyle CW, Brehm A, Bayer M, Solecka-Witulska B, Radford CE, Brown C, Nesbitt K, Dwyer C, Kannicht C, Spencer HT, Gaucher EA, Doering CB, Lillicrap D. Zakas PM, et al. Blood Adv. 2021 Feb 9;5(3):812-822. doi: 10.1182/bloodadvances.2020002971. Blood Adv. 2021. PMID: 33560395 Free PMC article.