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Page 1
Single-domain antibodies targeting antithrombin reduce bleeding in hemophilic mice with or without inhibitors.
Barbon E, Ayme G, Mohamadi A, Ottavi JF, Kawecki C, Casari C, Verhenne S, Marmier S, van Wittenberghe L, Charles S, Collaud F, Denis CV, Christophe OD, Mingozzi F, Lenting PJ. Barbon E, et al. Among authors: van wittenberghe l. EMBO Mol Med. 2020 Apr 7;12(4):e11298. doi: 10.15252/emmm.201911298. Epub 2020 Mar 11. EMBO Mol Med. 2020. PMID: 32159286 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: van wittenberghe l, van dijk r. 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: van wittenberghe l. 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: van wittenberghe l. 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: van wittenberghe l. 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.
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: van wittenberghe l. 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: van wittenberghe l. Nat Commun. 2018 Oct 5;9(1):4098. doi: 10.1038/s41467-018-06621-3. Nat Commun. 2018. PMID: 30291246 Free PMC article.
AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice.
Colella P, Sellier P, Costa Verdera H, Puzzo F, van Wittenberghe L, Guerchet N, Daniele N, Gjata B, Marmier S, Charles S, Simon Sola M, Ragone I, Leborgne C, Collaud F, Mingozzi F. Colella P, et al. Among authors: van wittenberghe l. Mol Ther Methods Clin Dev. 2018 Nov 17;12:85-101. doi: 10.1016/j.omtm.2018.11.002. eCollection 2019 Mar 15. Mol Ther Methods Clin Dev. 2018. PMID: 30581888 Free PMC article.
Capsid-specific removal of circulating antibodies to adeno-associated virus vectors.
Bertin B, Veron P, Leborgne C, Deschamps JY, Moullec S, Fromes Y, Collaud F, Boutin S, Latournerie V, van Wittenberghe L, Delache B, Le Grand R, Dereuddre-Bosquet N, Benveniste O, Moullier P, Masurier C, Merten O, Mingozzi F. Bertin B, et al. Among authors: van wittenberghe l. Sci Rep. 2020 Jan 21;10(1):864. doi: 10.1038/s41598-020-57893-z. Sci Rep. 2020. PMID: 31965041 Free PMC article.
26 results