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Liver-Directed Adeno-Associated Virus-Mediated Gene Therapy for Mucopolysaccharidosis Type VI.
Brunetti-Pierri N, Ferla R, Ginocchio VM, Rossi A, Fecarotta S, Romano R, Parenti G, Yildiz Y, Zancan S, Pecorella V, Dell'Anno M, Graziano M, Alliegro M, Andria G, Santamaria F, Brunetti-Pierri R, Simonelli F, Nigro V, Vargas M, Servillo G, Borgia F, Soscia E, Gargaro M, Funghini S, Tedesco N, Le Brun PR, Rupar CA, Prasad C, O'Callaghan M, Mitchell JJ, Danos O, Marteau JB, Galimberti S, Valsecchi MG, Veron P, Mingozzi F, Fallarino F, la Marca G, Sivri HS, Auricchio A. Brunetti-Pierri N, et al. Among authors: veron p. NEJM Evid. 2022 Jul;1(7):EVIDoa2200052. doi: 10.1056/EVIDoa2200052. Epub 2022 Jun 6. NEJM Evid. 2022. PMID: 38319253
Efficacy, pharmacokinetics, and safety in the mouse and primate retina of dual AAV vectors for Usher syndrome type 1B.
Ferla R, Dell'Aquila F, Doria M, Ferraiuolo M, Noto A, Grazioli F, Ammendola V, Testa F, Melillo P, Iodice C, Risca G, Tedesco N, le Brun PR, Surace EM, Simonelli F, Galimberti S, Valsecchi MG, Marteau JB, Veron P, Colloca S, Auricchio A. Ferla R, et al. Among authors: veron p. Mol Ther Methods Clin Dev. 2023 Feb 11;28:396-411. doi: 10.1016/j.omtm.2023.02.002. eCollection 2023 Mar 9. Mol Ther Methods Clin Dev. 2023. PMID: 36910588 Free PMC article.
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: veron p. Hum Gene Ther. 2019 Oct;30(10):1297-1305. doi: 10.1089/hum.2019.143. Hum Gene Ther. 2019. PMID: 31502485 Free PMC article.
Gene Therapy in Patients with the Crigler-Najjar Syndrome.
D'Antiga L, Beuers U, Ronzitti G, Brunetti-Pierri N, Baumann U, Di Giorgio A, Aronson S, Hubert A, Romano R, Junge N, Bosma P, Bortolussi G, Muro AF, Soumoudronga RF, Veron P, Collaud F, Knuchel-Legendre N, Labrune P, Mingozzi F. D'Antiga L, et al. Among authors: veron p. N Engl J Med. 2023 Aug 17;389(7):620-631. doi: 10.1056/NEJMoa2214084. N Engl J Med. 2023. PMID: 37585628 Clinical Trial.
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: veron p. 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.
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: veron p. Cell Immunol. 2019 Aug;342:103780. doi: 10.1016/j.cellimm.2018.03.004. Epub 2018 Mar 16. Cell Immunol. 2019. PMID: 29571923
Analysis of Adeno-Associated Virus Serotype 8 (AAV8)-antibody complexes using epitope mapping by molecular imprinting leads to the identification of Fab peptides that potentially evade AAV8 neutralisation.
Piletska E, Veron P, Bertin B, Mingozzi F, Jones D, Norman RL, Earley J, Karim K, Garcia-Cruz A, Piletsky S. Piletska E, et al. Among authors: veron p. Nanomedicine. 2023 Aug;52:102691. doi: 10.1016/j.nano.2023.102691. Epub 2023 Jun 15. Nanomedicine. 2023. PMID: 37329939 Free article.
50 results