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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: mingozzi f. 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.
Treatment of infantile-onset Pompe disease in a rat model with muscle-directed AAV gene therapy.
Muñoz S, Bertolin J, Jimenez V, Jaén ML, Garcia M, Pujol A, Vilà L, Sacristan V, Barbon E, Ronzitti G, El Andari J, Tulalamba W, Pham QH, Ruberte J, VandenDriessche T, Chuah MK, Grimm D, Mingozzi F, Bosch F. Muñoz S, et al. Among authors: mingozzi f. Mol Metab. 2024 Mar;81:101899. doi: 10.1016/j.molmet.2024.101899. Epub 2024 Feb 10. Mol Metab. 2024. PMID: 38346589 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: mingozzi f. 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: mingozzi f. Nanomedicine. 2023 Aug;52:102691. doi: 10.1016/j.nano.2023.102691. Epub 2023 Jun 15. Nanomedicine. 2023. PMID: 37329939 Free article.
Muscle-specific, liver-detargeted adeno-associated virus gene therapy rescues Pompe phenotype in adult and neonate Gaa-/- mice.
Sellier P, Vidal P, Bertin B, Gicquel E, Bertil-Froidevaux E, Georger C, van Wittenberghe L, Miranda A, Daniele N, Richard I, Gross DA, Mingozzi F, Collaud F, Ronzitti G. Sellier P, et al. Among authors: mingozzi f. J Inherit Metab Dis. 2024 Jan;47(1):119-134. doi: 10.1002/jimd.12625. Epub 2023 May 29. J Inherit Metab Dis. 2024. PMID: 37204237
Successful treatment of severe MSUD in Bckdhb-/- mice with neonatal AAV gene therapy.
Pontoizeau C, Gaborit C, Tual N, Simon-Sola M, Rotaru I, Benoist M, Colella P, Lamazière A, Brassier A, Arnoux JB, Rötig A, Ottolenghi C, de Lonlay P, Mingozzi F, Cavazzana M, Schiff M. Pontoizeau C, et al. Among authors: mingozzi f. J Inherit Metab Dis. 2024 Jan;47(1):41-49. doi: 10.1002/jimd.12604. Epub 2023 Mar 15. J Inherit Metab Dis. 2024. PMID: 36880392
175 results