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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: mingozzi f. 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 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: mingozzi 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.
Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs.
Mack DL, Poulard K, Goddard MA, Latournerie V, Snyder JM, Grange RW, Elverman MR, Denard J, Veron P, Buscara L, Le Bec C, Hogrel JY, Brezovec AG, Meng H, Yang L, Liu F, O'Callaghan M, Gopal N, Kelly VE, Smith BK, Strande JL, Mavilio F, Beggs AH, Mingozzi F, Lawlor MW, Buj-Bello A, Childers MK. Mack DL, et al. Among authors: mingozzi f. Mol Ther. 2017 Apr 5;25(4):839-854. doi: 10.1016/j.ymthe.2017.02.004. Epub 2017 Feb 22. Mol Ther. 2017. PMID: 28237839 Free PMC article.
Autophagy determines efficiency of liver-directed gene therapy with adeno-associated viral vectors.
Hösel M, Huber A, Bohlen S, Lucifora J, Ronzitti G, Puzzo F, Boisgerault F, Hacker UT, Kwanten WJ, Klöting N, Blüher M, Gluschko A, Schramm M, Utermöhlen O, Bloch W, Mingozzi F, Krut O, Büning H. Hösel M, et al. Among authors: mingozzi f. Hepatology. 2017 Jul;66(1):252-265. doi: 10.1002/hep.29176. Epub 2017 May 29. Hepatology. 2017. PMID: 28318036 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: mingozzi f. Sci Transl Med. 2017 Nov 29;9(418):eaam6375. doi: 10.1126/scitranslmed.aam6375. Sci Transl Med. 2017. PMID: 29187643 Free PMC article.
Combination Therapy Is the New Gene Therapy?
Ronzitti G, Mingozzi F. Ronzitti G, et al. Among authors: mingozzi f. Mol Ther. 2018 Jan 3;26(1):12-14. doi: 10.1016/j.ymthe.2017.12.008. Epub 2017 Dec 20. Mol Ther. 2018. PMID: 29273499 Free PMC article. No abstract available.
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: mingozzi 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.
174 results