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Page 1
Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy.
Deichmann A, Hacein-Bey-Abina S, Schmidt M, Garrigue A, Brugman MH, Hu J, Glimm H, Gyapay G, Prum B, Fraser CC, Fischer N, Schwarzwaelder K, Siegler ML, de Ridder D, Pike-Overzet K, Howe SJ, Thrasher AJ, Wagemaker G, Abel U, Staal FJ, Delabesse E, Villeval JL, Aronow B, Hue C, Prinz C, Wissler M, Klanke C, Weissenbach J, Alexander I, Fischer A, von Kalle C, Cavazzana-Calvo M. Deichmann A, et al. Among authors: alexander i. J Clin Invest. 2007 Aug;117(8):2225-32. doi: 10.1172/JCI31659. J Clin Invest. 2007. PMID: 17671652 Free PMC article.
LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1.
Hacein-Bey-Abina S, Von Kalle C, Schmidt M, McCormack MP, Wulffraat N, Leboulch P, Lim A, Osborne CS, Pawliuk R, Morillon E, Sorensen R, Forster A, Fraser P, Cohen JI, de Saint Basile G, Alexander I, Wintergerst U, Frebourg T, Aurias A, Stoppa-Lyonnet D, Romana S, Radford-Weiss I, Gross F, Valensi F, Delabesse E, Macintyre E, Sigaux F, Soulier J, Leiva LE, Wissler M, Prinz C, Rabbitts TH, Le Deist F, Fischer A, Cavazzana-Calvo M. Hacein-Bey-Abina S, et al. Among authors: alexander i. Science. 2003 Oct 17;302(5644):415-9. doi: 10.1126/science.1088547. Science. 2003. PMID: 14564000
Treatment of an infant with X-linked severe combined immunodeficiency (SCID-X1) by gene therapy in Australia.
Ginn SL, Curtin JA, Kramer B, Smyth CM, Wong M, Kakakios A, McCowage GB, Watson D, Alexander SI, Latham M, Cunningham SC, Zheng M, Hobson L, Rowe PB, Fischer A, Cavazzana-Calvo M, Hacein-Bey-Abina S, Alexander IE. Ginn SL, et al. Among authors: alexander si, alexander ie. Med J Aust. 2005 May 2;182(9):458-63. doi: 10.5694/j.1326-5377.2005.tb06785.x. Med J Aust. 2005. PMID: 15865589
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. 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.
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. 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.
AAV-p40 Bioengineering Platform for Variant Selection Based on Transgene Expression.
Westhaus A, Cabanes-Creus M, Jonker T, Sallard E, Navarro RG, Zhu E, Baltazar Torres G, Lee S, Wilmott P, Gonzalez-Cordero A, Santilli G, Thrasher AJ, Alexander IE, Lisowski L. Westhaus A, et al. Hum Gene Ther. 2022 Jun;33(11-12):664-682. doi: 10.1089/hum.2021.278. Epub 2022 May 6. Hum Gene Ther. 2022. PMID: 35297686 Free PMC article.
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.
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. Sci Transl Med. 2020 Sep 9;12(560):eaba3312. doi: 10.1126/scitranslmed.aba3312. Sci Transl Med. 2020. PMID: 32908003
Safety and efficacy of an engineered hepatotropic AAV gene therapy for ornithine transcarbamylase deficiency in cynomolgus monkeys.
Baruteau J, Cunningham SC, Yilmaz BS, Perocheau DP, Eaglestone S, Burke D, Thrasher AJ, Waddington SN, Lisowski L, Alexander IE, Gissen P. Baruteau J, et al. Mol Ther Methods Clin Dev. 2021 Sep 14;23:135-146. doi: 10.1016/j.omtm.2021.09.005. eCollection 2021 Dec 10. Mol Ther Methods Clin Dev. 2021. PMID: 34703837 Free PMC article.
393 results