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Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome.
Collaud F, Bortolussi G, Guianvarc'h L, Aronson SJ, Bordet T, Veron P, Charles S, Vidal P, Sola MS, Rundwasser S, Dufour DG, Lacoste F, Luc C, Wittenberghe LV, Martin S, Le Bec C, Bosma PJ, Muro AF, Ronzitti G, Hebben M, Mingozzi F. Collaud F, et al. Among authors: aronson sj. Mol Ther Methods Clin Dev. 2018 Dec 31;12:157-174. doi: 10.1016/j.omtm.2018.12.011. eCollection 2019 Mar 15. Mol Ther Methods Clin Dev. 2018. PMID: 30705921 Free PMC article.
Prevalence and Relevance of Pre-Existing Anti-Adeno-Associated Virus Immunity in the Context of Gene Therapy for Crigler-Najjar Syndrome.
Aronson SJ, Veron P, Collaud F, Hubert A, Delahais V, Honnet G, de Knegt RJ, Junge N, Baumann U, Di Giorgio A, D'Antiga L, Ginocchio VM, Brunetti-Pierri N, Labrune P, Beuers U, Bosma PJ, Mingozzi F. Aronson SJ, et al. Hum Gene Ther. 2019 Oct;30(10):1297-1305. doi: 10.1089/hum.2019.143. Hum Gene Ther. 2019. PMID: 31502485 Free PMC article.
A Quantitative In Vitro Potency Assay for Adeno-Associated Virus Vectors Encoding for the UGT1A1 Transgene.
Aronson SJ, Bakker RS, Moenis S, van Dijk R, Bortolussi G, Collaud F, Shi X, Duijst S, Ten Bloemendaal L, Ronzitti G, Muro AF, Mingozzi F, Beuers U, Bosma PJ. Aronson SJ, et al. Mol Ther Methods Clin Dev. 2020 Jun 3;18:250-258. doi: 10.1016/j.omtm.2020.06.002. eCollection 2020 Sep 11. Mol Ther Methods Clin Dev. 2020. PMID: 32637454 Free PMC article.
Efficacy of AAV8-hUGT1A1 with Rapamycin in neonatal, suckling, and juvenile rats to model treatment in pediatric CNs patients.
Shi X, Aronson SJ, Ten Bloemendaal L, Duijst S, Bakker RS, de Waart DR, Bortolussi G, Collaud F, Oude Elferink RP, Muro AF, Mingozzi F, Ronzitti G, Bosma PJ. Shi X, et al. Among authors: aronson sj. Mol Ther Methods Clin Dev. 2020 Dec 3;20:287-297. doi: 10.1016/j.omtm.2020.11.016. eCollection 2021 Mar 12. Mol Ther Methods Clin Dev. 2020. PMID: 33511243 Free PMC article.
Disease burden and management of Crigler-Najjar syndrome: Report of a world registry.
Aronson SJ, Junge N, Trabelsi M, Kelmemi W, Hubert A, Brigatti KW, Fox MD, de Knegt RJ, Escher JC, Ginocchio VM, Iorio R, Zhu Y, Özçay F, Rahim F, El-Shabrawi MHF, Shteyer E, Di Giorgio A, D'Antiga L, Mingozzi F, Brunetti-Pierri N, Strauss KA, Labrune P, Mrad R, Baumann U, Beuers U, Bosma PJ; CureCN Consortium. Aronson SJ, et al. Liver Int. 2022 Jul;42(7):1593-1604. doi: 10.1111/liv.15239. Epub 2022 Mar 29. Liver Int. 2022. PMID: 35274801 No abstract available.
What's next in gene therapy for Crigler-Najjar syndrome?
Aronson SJ, Ronzitti G, Bosma PJ. Aronson SJ, et al. Expert Opin Biol Ther. 2023 Feb;23(2):119-121. doi: 10.1080/14712598.2022.2160237. Epub 2022 Dec 29. Expert Opin Biol Ther. 2023. PMID: 36579791 No abstract available.
35 results