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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: alexander ie. 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.
Coherence analysis discriminates between retroviral integration patterns in CD34(+) cells transduced under differing clinical trial conditions.
Hallwirth CV, Garg G, Peters TJ, Kramer BA, Malani NV, Hyman J, Ruan X, Ginn SL, Hetherington NA, Veeravalli L, Shahab A, Ranganathan S, Wei CL, Liddle C, Thrasher AJ, Bushman FD, Buckley MJ, Alexander IE. Hallwirth CV, et al. Among authors: alexander ie. Mol Ther Methods Clin Dev. 2015 Apr 29;2:15015. doi: 10.1038/mtm.2015.15. eCollection 2015. Mol Ther Methods Clin Dev. 2015. PMID: 26029726 Free PMC article.
Adeno-associated virus serotypes for gene therapeutics.
Lisowski L, Tay SS, Alexander IE. Lisowski L, et al. Among authors: alexander ie. Curr Opin Pharmacol. 2015 Oct;24:59-67. doi: 10.1016/j.coph.2015.07.006. Epub 2015 Aug 25. Curr Opin Pharmacol. 2015. PMID: 26291407 Review.
Limiting Thymic Precursor Supply Increases the Risk of Lymphoid Malignancy in Murine X-Linked Severe Combined Immunodeficiency.
Ginn SL, Hallwirth CV, Liao SHY, Teber ET, Arthur JW, Wu J, Lee HC, Tay SS, Hu M, Reddel RR, McCormack MP, Thrasher AJ, Cavazzana M, Alexander SI, Alexander IE. Ginn SL, et al. Among authors: alexander si, alexander ie. Mol Ther Nucleic Acids. 2017 Mar 17;6:1-14. doi: 10.1016/j.omtn.2016.11.011. Epub 2016 Dec 10. Mol Ther Nucleic Acids. 2017. PMID: 28325276 Free PMC article.
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: alexander ie. 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: alexander ie. 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.
Gene therapy clinical trials worldwide to 2017: An update.
Ginn SL, Amaya AK, Alexander IE, Edelstein M, Abedi MR. Ginn SL, et al. Among authors: alexander ie. J Gene Med. 2018 May;20(5):e3015. doi: 10.1002/jgm.3015. Epub 2018 Apr 19. J Gene Med. 2018. PMID: 29575374 Review.
189 results