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Page 1
Gene Therapy in Patients with the Crigler-Najjar Syndrome.
D'Antiga L, Beuers U, Ronzitti G, Brunetti-Pierri N, Baumann U, Di Giorgio A, Aronson S, Hubert A, Romano R, Junge N, Bosma P, Bortolussi G, Muro AF, Soumoudronga RF, Veron P, Collaud F, Knuchel-Legendre N, Labrune P, Mingozzi F. D'Antiga L, et al. Among authors: collaud f. N Engl J Med. 2023 Aug 17;389(7):620-631. doi: 10.1056/NEJMoa2214084. N Engl J Med. 2023. PMID: 37585628 Clinical Trial.
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. Among authors: collaud f. 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 translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome.
Ronzitti G, Bortolussi G, van Dijk R, Collaud F, Charles S, Leborgne C, Vidal P, Martin S, Gjata B, Sola MS, van Wittenberghe L, Vignaud A, Veron P, Bosma PJ, Muro AF, Mingozzi F. Ronzitti G, et al. Among authors: collaud f. Mol Ther Methods Clin Dev. 2016 Jul 20;3:16049. doi: 10.1038/mtm.2016.49. eCollection 2016. Mol Ther Methods Clin Dev. 2016. PMID: 27722180 Free PMC article.
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. 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.
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. Among authors: collaud f. 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: collaud f. 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.
Progress and challenges of gene therapy for Pompe disease.
Ronzitti G, Collaud F, Laforet P, Mingozzi F. Ronzitti G, et al. Among authors: collaud f. Ann Transl Med. 2019 Jul;7(13):287. doi: 10.21037/atm.2019.04.67. Ann Transl Med. 2019. PMID: 31392199 Free PMC article. Review.
Long-term correction of ornithine transcarbamylase deficiency in Spf-Ash mice with a translationally optimized AAV vector.
De Sabbata G, Boisgerault F, Guarnaccia C, Iaconcig A, Bortolussi G, Collaud F, Ronzitti G, Sola MS, Vidal P, Rouillon J, Charles S, Nicastro E, D'Antiga L, Ilyinskii P, Mingozzi F, Kishimoto TK, Muro AF. De Sabbata G, et al. Among authors: collaud f. Mol Ther Methods Clin Dev. 2020 Nov 17;20:169-180. doi: 10.1016/j.omtm.2020.11.005. eCollection 2021 Mar 12. Mol Ther Methods Clin Dev. 2020. PMID: 33473356 Free PMC article.
Exposure to wild-type AAV drives distinct capsid immunity profiles in humans.
Kuranda K, Jean-Alphonse P, Leborgne C, Hardet R, Collaud F, Marmier S, Costa Verdera H, Ronzitti G, Veron P, Mingozzi F. Kuranda K, et al. Among authors: collaud f. J Clin Invest. 2018 Dec 3;128(12):5267-5279. doi: 10.1172/JCI122372. Epub 2018 Oct 22. J Clin Invest. 2018. PMID: 30352429 Free PMC article.
IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies.
Leborgne C, Barbon E, Alexander JM, Hanby H, Delignat S, Cohen DM, Collaud F, Muraleetharan S, Lupo D, Silverberg J, Huang K, van Wittengerghe L, Marolleau B, Miranda A, Fabiano A, Daventure V, Beck H, Anguela XM, Ronzitti G, Armour SM, Lacroix-Desmazes S, Mingozzi F. Leborgne C, et al. Among authors: collaud f. Nat Med. 2020 Jul;26(7):1096-1101. doi: 10.1038/s41591-020-0911-7. Epub 2020 Jun 1. Nat Med. 2020. PMID: 32483358 Free article.
29 results