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Page 1
Muscle-specific, liver-detargeted adeno-associated virus gene therapy rescues Pompe phenotype in adult and neonate Gaa-/- mice.
Sellier P, Vidal P, Bertin B, Gicquel E, Bertil-Froidevaux E, Georger C, van Wittenberghe L, Miranda A, Daniele N, Richard I, Gross DA, Mingozzi F, Collaud F, Ronzitti G. Sellier P, et al. Among authors: collaud f. J Inherit Metab Dis. 2024 Jan;47(1):119-134. doi: 10.1002/jimd.12625. Epub 2023 May 29. J Inherit Metab Dis. 2024. PMID: 37204237
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.
Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase.
Puzzo F, Colella P, Biferi MG, Bali D, Paulk NK, Vidal P, Collaud F, Simon-Sola M, Charles S, Hardet R, Leborgne C, Meliani A, Cohen-Tannoudji M, Astord S, Gjata B, Sellier P, van Wittenberghe L, Vignaud A, Boisgerault F, Barkats M, Laforet P, Kay MA, Koeberl DD, Ronzitti G, Mingozzi F. Puzzo F, et al. Among authors: collaud f. Sci Transl Med. 2017 Nov 29;9(418):eaam6375. doi: 10.1126/scitranslmed.aam6375. Sci Transl Med. 2017. PMID: 29187643 Free PMC article.
Enhanced liver gene transfer and evasion of preexisting humoral immunity with exosome-enveloped AAV vectors.
Meliani A, Boisgerault F, Fitzpatrick Z, Marmier S, Leborgne C, Collaud F, Simon Sola M, Charles S, Ronzitti G, Vignaud A, van Wittenberghe L, Marolleau B, Jouen F, Tan S, Boyer O, Christophe O, Brisson AR, Maguire CA, Mingozzi F. Meliani A, et al. Among authors: collaud f. Blood Adv. 2017 Oct 16;1(23):2019-2031. doi: 10.1182/bloodadvances.2017010181. eCollection 2017 Oct 24. Blood Adv. 2017. PMID: 29296848 Free PMC article.
Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression.
Vidal P, Pagliarani S, Colella P, Costa Verdera H, Jauze L, Gjorgjieva M, Puzzo F, Marmier S, Collaud F, Simon Sola M, Charles S, Lucchiari S, van Wittenberghe L, Vignaud A, Gjata B, Richard I, Laforet P, Malfatti E, Mithieux G, Rajas F, Comi GP, Ronzitti G, Mingozzi F. Vidal P, et al. Among authors: collaud f. Mol Ther. 2018 Mar 7;26(3):890-901. doi: 10.1016/j.ymthe.2017.12.019. Epub 2017 Dec 28. Mol Ther. 2018. PMID: 29396266 Free PMC article.
Prevalence and long-term monitoring of humoral immunity against adeno-associated virus in Duchenne Muscular Dystrophy patients.
Leborgne C, Latournerie V, Boutin S, Desgue D, Quéré A, Pignot E, Collaud F, Charles S, Simon Sola M, Masat E, Jouen F, Boyer O, Masurier C, Mingozzi F, Veron P. Leborgne C, et al. Among authors: collaud f. Cell Immunol. 2019 Aug;342:103780. doi: 10.1016/j.cellimm.2018.03.004. Epub 2018 Mar 16. Cell Immunol. 2019. PMID: 29571923
Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction.
Fitzpatrick Z, Leborgne C, Barbon E, Masat E, Ronzitti G, van Wittenberghe L, Vignaud A, Collaud F, Charles S, Simon Sola M, Jouen F, Boyer O, Mingozzi F. Fitzpatrick Z, et al. Among authors: collaud f. Mol Ther Methods Clin Dev. 2018 Feb 13;9:119-129. doi: 10.1016/j.omtm.2018.02.003. eCollection 2018 Jun 15. Mol Ther Methods Clin Dev. 2018. PMID: 29766022 Free PMC article.
Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration.
Meliani A, Boisgerault F, Hardet R, Marmier S, Collaud F, Ronzitti G, Leborgne C, Costa Verdera H, Simon Sola M, Charles S, Vignaud A, van Wittenberghe L, Manni G, Christophe O, Fallarino F, Roy C, Michaud A, Ilyinskii P, Kishimoto TK, Mingozzi F. Meliani A, et al. Among authors: collaud f. Nat Commun. 2018 Oct 5;9(1):4098. doi: 10.1038/s41467-018-06621-3. Nat Commun. 2018. PMID: 30291246 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.
30 results