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Recapitulation of Skewed X-Inactivation in Female Ornithine Transcarbamylase-Deficient Primary Human Hepatocytes in the FRG Mouse: A Novel System for Developing Epigenetic Therapies.
Cunningham SC, van Dijk EB, Zhu E, Sugden M, Mandwie M, Siew S, Devanapalli B, Tolun AA, Klein A, Wilson L, Aryamanesh N, Gissen P, Baruteau J, Bhattacharya K, Alexander IE. Cunningham SC, et al. Among authors: van dijk eb. Hum Gene Ther. 2023 Sep;34(17-18):917-926. doi: 10.1089/hum.2023.011. Epub 2023 Sep 11. Hum Gene Ther. 2023. PMID: 37350098
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. Among authors: van dijk eb. 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