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Repeated dosing of AAV-mediated liver gene therapy in juvenile rat and mouse models of Crigler-Najjar syndrome type I.
Shi X, Bortolussi G, Collaud F, Lebrun PR, Bloemendaal LT, Guerchet N, Rudi de Waart D, Sellier P, Duijst S, Veron P, Mingozzi F, Kishimoto TK, Ronzitti G, Bosma P, Muro AF. Shi X, et al. Among authors: veron p. Mol Ther Methods Clin Dev. 2024 Oct 28;32(4):101363. doi: 10.1016/j.omtm.2024.101363. eCollection 2024 Dec 12. Mol Ther Methods Clin Dev. 2024. PMID: 39618425 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.
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.
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.
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 Clinical Trial.
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: veron p. Sci Rep. 2020 Jan 21;10(1):864. doi: 10.1038/s41598-020-57893-z. Sci Rep. 2020. PMID: 31965041 Free PMC article.
52 results