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Page 1
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.
Short-lived recombinant adeno-associated virus transgene expression in dystrophic muscle is associated with oxidative damage to transgene mRNA.
Dupont JB, Tournaire B, Georger C, Marolleau B, Jeanson-Leh L, Ledevin M, Lindenbaum P, Lecomte E, Cogné B, Dubreil L, Larcher T, Gjata B, Van Wittenberghe L, Le Guiner C, Penaud-Budloo M, Snyder RO, Moullier P, Léger A. Dupont JB, et al. Among authors: van wittenberghe l. Mol Ther Methods Clin Dev. 2015 Apr 8;2:15010. doi: 10.1038/mtm.2015.10. eCollection 2015. Mol Ther Methods Clin Dev. 2015. PMID: 26029721 Free PMC article.
Efficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophy.
Armbruster N, Lattanzi A, Jeavons M, Van Wittenberghe L, Gjata B, Marais T, Martin S, Vignaud A, Voit T, Mavilio F, Barkats M, Buj-Bello A. Armbruster N, et al. Among authors: van wittenberghe l. Mol Ther Methods Clin Dev. 2016 Sep 14;3:16060. doi: 10.1038/mtm.2016.60. eCollection 2016. Mol Ther Methods Clin Dev. 2016. PMID: 27652289 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.
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.
27 results