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Liver-specific transcriptional modules identified by genome-wide in silico analysis enable efficient gene therapy in mice and non-human primates.
Chuah MK, Petrus I, De Bleser P, Le Guiner C, Gernoux G, Adjali O, Nair N, Willems J, Evens H, Rincon MY, Matrai J, Di Matteo M, Samara-Kuko E, Yan B, Acosta-Sanchez A, Meliani A, Cherel G, Blouin V, Christophe O, Moullier P, Mingozzi F, VandenDriessche T. Chuah MK, et al. Among authors: mingozzi f. Mol Ther. 2014 Sep;22(9):1605-13. doi: 10.1038/mt.2014.114. Epub 2014 Jun 23. Mol Ther. 2014. PMID: 24954473 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.
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: mingozzi f. Cell Immunol. 2019 Aug;342:103780. doi: 10.1016/j.cellimm.2018.03.004. Epub 2018 Mar 16. Cell Immunol. 2019. PMID: 29571923
Gene Therapy for Pompe Disease: The Time is now.
Colella P, Mingozzi F. Colella P, et al. Among authors: mingozzi f. Hum Gene Ther. 2019 Oct;30(10):1245-1262. doi: 10.1089/hum.2019.109. Epub 2019 Sep 9. Hum Gene Ther. 2019. PMID: 31298581 Review.
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: mingozzi 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.
175 results