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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: zhu e. 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
Identification of liver-specific enhancer-promoter activity in the 3' untranslated region of the wild-type AAV2 genome.
Logan GJ, Dane AP, Hallwirth CV, Smyth CM, Wilkie EE, Amaya AK, Zhu E, Khandekar N, Ginn SL, Liao SHY, Cunningham SC, Sasaki N, Cabanes-Creus M, Tam PPL, Russell DW, Lisowski L, Alexander IE. Logan GJ, et al. Among authors: zhu e. Nat Genet. 2017 Aug;49(8):1267-1273. doi: 10.1038/ng.3893. Epub 2017 Jun 19. Nat Genet. 2017. PMID: 28628105
Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity.
Paulk NK, Pekrun K, Zhu E, Nygaard S, Li B, Xu J, Chu K, Leborgne C, Dane AP, Haft A, Zhang Y, Zhang F, Morton C, Valentine MB, Davidoff AM, Nathwani AC, Mingozzi F, Grompe M, Alexander IE, Lisowski L, Kay MA. Paulk NK, et al. Among authors: zhu e. Mol Ther. 2018 Jan 3;26(1):289-303. doi: 10.1016/j.ymthe.2017.09.021. Epub 2017 Sep 25. Mol Ther. 2018. PMID: 29055620 Free PMC article.
High-Throughput In Vitro, Ex Vivo, and In Vivo Screen of Adeno-Associated Virus Vectors Based on Physical and Functional Transduction.
Westhaus A, Cabanes-Creus M, Rybicki A, Baltazar G, Navarro RG, Zhu E, Drouyer M, Knight M, Albu RF, Ng BH, Kalajdzic P, Kwiatek M, Hsu K, Santilli G, Gold W, Kramer B, Gonzalez-Cordero A, Thrasher AJ, Alexander IE, Lisowski L. Westhaus A, et al. Among authors: zhu e. Hum Gene Ther. 2020 May;31(9-10):575-589. doi: 10.1089/hum.2019.264. Epub 2020 Feb 26. Hum Gene Ther. 2020. PMID: 32000541 Free PMC article.
Efficient in vivo editing of OTC-deficient patient-derived primary human hepatocytes.
Ginn SL, Amaya AK, Liao SHY, Zhu E, Cunningham SC, Lee M, Hallwirth CV, Logan GJ, Tay SS, Cesare AJ, Pickett HA, Grompe M, Dilworth K, Lisowski L, Alexander IE. Ginn SL, et al. Among authors: zhu e. JHEP Rep. 2019 Dec 27;2(1):100065. doi: 10.1016/j.jhepr.2019.100065. eCollection 2020 Feb. JHEP Rep. 2019. PMID: 32039406 Free PMC article.
Attenuation of Heparan Sulfate Proteoglycan Binding Enhances In Vivo Transduction of Human Primary Hepatocytes with AAV2.
Cabanes-Creus M, Westhaus A, Navarro RG, Baltazar G, Zhu E, Amaya AK, Liao SHY, Scott S, Sallard E, Dilworth KL, Rybicki A, Drouyer M, Hallwirth CV, Bennett A, Santilli G, Thrasher AJ, Agbandje-McKenna M, Alexander IE, Lisowski L. Cabanes-Creus M, et al. Among authors: zhu e. Mol Ther Methods Clin Dev. 2020 May 13;17:1139-1154. doi: 10.1016/j.omtm.2020.05.004. eCollection 2020 Jun 12. Mol Ther Methods Clin Dev. 2020. PMID: 32490035 Free PMC article.
Restoring the natural tropism of AAV2 vectors for human liver.
Cabanes-Creus M, Hallwirth CV, Westhaus A, Ng BH, Liao SHY, Zhu E, Navarro RG, Baltazar G, Drouyer M, Scott S, Logan GJ, Santilli G, Bennett A, Ginn SL, McCaughan G, Thrasher AJ, Agbandje-McKenna M, Alexander IE, Lisowski L. Cabanes-Creus M, et al. Among authors: zhu e. Sci Transl Med. 2020 Sep 9;12(560):eaba3312. doi: 10.1126/scitranslmed.aba3312. Sci Transl Med. 2020. PMID: 32908003
Single amino acid insertion allows functional transduction of murine hepatocytes with human liver tropic AAV capsids.
Cabanes-Creus M, Navarro RG, Liao SHY, Baltazar G, Drouyer M, Zhu E, Scott S, Luong C, Wilson LOW, Alexander IE, Lisowski L. Cabanes-Creus M, et al. Among authors: zhu e. Mol Ther Methods Clin Dev. 2021 Apr 24;21:607-620. doi: 10.1016/j.omtm.2021.04.010. eCollection 2021 Jun 11. Mol Ther Methods Clin Dev. 2021. PMID: 34095344 Free PMC article.
Novel human liver-tropic AAV variants define transferable domains that markedly enhance the human tropism of AAV7 and AAV8.
Cabanes-Creus M, Navarro RG, Zhu E, Baltazar G, Liao SHY, Drouyer M, Amaya AK, Scott S, Nguyen LH, Westhaus A, Hebben M, Wilson LOW, Thrasher AJ, Alexander IE, Lisowski L. Cabanes-Creus M, et al. Among authors: zhu e. Mol Ther Methods Clin Dev. 2021 Nov 25;24:88-101. doi: 10.1016/j.omtm.2021.11.011. eCollection 2022 Mar 10. Mol Ther Methods Clin Dev. 2021. PMID: 34977275 Free PMC article.
554 results