Adeno-Associated Virus Vector-Mediated Liver Gene Therapy for Crigler-Najjar Syndrome

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Publications

Capsid-specific removal of circulating antibodies to adeno-associated virus vectors

Auteurs: Berangere Bertin, Philippe Veron, Christian Leborgne, Jack-Yves Deschamps, Sophie Moullec, Yves Fromes, Fanny Collaud, Sylvie Boutin, Virginie Latournerie, Laetitia van Wittenberghe, Benoit Delache, Roger Le Grand, Nathalie Dereuddre-Bosquet, Olivier Benveniste, Philippe Moullier, Carole Masurier, Otto Merten, Federico Mingozzi
Publié dans: Scientific Reports, Numéro 10/1, 2020, ISSN 2045-2322
Éditeur: Nature Publishing Group
DOI: 10.1038/s41598-020-57893-z

A Quantitative In Vitro Potency Assay for Adeno-Associated Virus Vectors Encoding for the UGT1A1 Transgene

Auteurs: Sem J. Aronson, Robert S. Bakker, Sascha Moenis, Remco van Dijk, Giulia Bortolussi, Fanny Collaud, Xiaoxia Shi, Suzanne Duijst, Lysbeth ten Bloemendaal, Giuseppe Ronzitti, Andrés F. Muro, Federico Mingozzi, Ulrich Beuers, Piter J. Bosma
Publié dans: Molecular Therapy - Methods & Clinical Development, Numéro 18, 2020, Page(s) 250-258, ISSN 2329-0501
Éditeur: Elsevier
DOI: 10.1016/j.omtm.2020.06.002

Diagnosis and management of secondary causes of steatohepatitis

Auteurs: R. Liebe; Irene Esposito; Hans H. Bock; Stephan vom Dahl; Jan Stindt; Ulrich Baumann; Ulrich Baumann; Tom Luedde; Verena Keitel
Publié dans: Journal of Hepatology, Numéro 74(6), 2021, Page(s) 1455-1471, ISSN 1600-0641
Éditeur: Elsevier
DOI: 10.1016/j.jhep.2021.01.045

Disease burden and management of <scp>Crigler‐Najjar</scp> syndrome: Report of a world registry

Auteurs: Robert De Knegt
Publié dans: Liver International, Numéro 14783223, 2022, Page(s) 1593-1604, ISSN 1478-3223
Éditeur: Blackwell Publishing Inc.
DOI: 10.1111/liv.15239

Evading and overcoming AAV neutralization in gene therapy

Auteurs: Joseph Earley, Elena Piletska, Giuseppe Ronzitti, Sergey Piletsky
Publié dans: Trends in Biotechnology, Numéro Volume 41, Numéro 6, 2023, Page(s) 836-845, ISSN 0167-7799
Éditeur: Elsevier BV
DOI: 10.1016/j.tibtech.2022.11.006

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

Auteurs: Elena Piletska, Philippe Veron, Bérangère Bertin, Federico Mingozzi, Donald Jones, Rachel L. Norman, Joseph Earley, Kal Karim, Alvaro Garcia-Cruz, Sergey Piletsky
Publié dans: Nanomedicine: Nanotechnology, Biology, and Medicine, Numéro Volume 52, August 2023, 2023, Page(s) 1-10, ISSN 2322-5904
Éditeur: Nanomedicine: Nanotechnology, Biology, and Medicine
DOI: 10.1016/j.nano.2023.102691

Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome

Auteurs: Fanny Collaud, Giulia Bortolussi, Laurence Guianvarc’h, Sem J. Aronson, Thierry Bordet, Philippe Veron, Severine Charles, Patrice Vidal, Marcelo Simon Sola, Stephanie Rundwasser, Delphine G. Dufour, Florence Lacoste, Cyril Luc, Laetitia v. Wittenberghe, Samia Martin, Christine Le Bec, Piter J. Bosma, Andres F. Muro, Giuseppe Ronzitti, Matthias Hebben, Federico Mingozzi
Publié dans: Molecular Therapy - Methods & Clinical Development, Numéro 12, 2019, Page(s) 157-174, ISSN 2329-0501
Éditeur: Elsevier
DOI: 10.1016/j.omtm.2018.12.011

Gene Therapy in Patients with the Crigler–Najjar Syndrome

Auteurs: Lorenzo D’Antiga; Ulrich Beuers; Giuseppe Ronzitti; Nicola Brunetti-Pierri; Ulrich Baumann; Angelo Di Giorgio; Sem Aronson; Aurelie Hubert; Roberta Romano; Norman Junge; Piter Bosma; Giulia Bortolussi; Andrés F. Muro; Ravaka F. Soumoudronga; Philippe Veron; Fanny Collaud; Nathalie Knuchel-Legendre; Philippe Labrune; Federico Mingozzi
Publié dans: https://hal.science/hal-04324294, Numéro 389, 2023, Page(s) 620-631, ISSN 1533-4406
Éditeur: The New England Journal of Medicine
DOI: 10.1056/nejmoa2214084

Prevalence and Relevance of Pre-Existing Anti-Adeno-Associated Virus Immunity in the Context of Gene Therapy for Crigler–Najjar Syndrome

Auteurs: Sem J. Aronson, Philippe Veron, Fanny Collaud, Aurélie Hubert, Virginie Delahais, Géraldine Honnet, Robert J. de Knegt, Norman Junge, Ulrich Baumann, Angelo Di Giorgio, Lorenzo D'Antiga, Virginia M. Ginocchio, Nicola Brunetti-Pierri, Philippe Labrune, Ulrich Beuers, Piter J. Bosma, Federico Mingozzi
Publié dans: Human Gene Therapy, Numéro 30/10, 2019, Page(s) 1297-1305, ISSN 1043-0342
Éditeur: Mary Ann Liebert Inc.
DOI: 10.1089/hum.2019.143

Quality of Life in patients with Crigler-Najjar Syndrome

Auteurs: Junge, Aronson, Baumann, Bosma, D´Antiga, Di Giorgio, Taibi, Labrune
Publié dans: ESPGHAN Concgress 2024, 2024
Éditeur: ESPGHAN Concgress 2024

Natural history and current management of Crigler-Najjar syndrome: report of the CN World Registry

Auteurs: Sem Aronson, Wided Kelmemi, Madiha Trabelsi, Aurélie Hubert, Karlla W. Brigatti, Michael D. Fox, Dr. Robert J. De Knegt, Johanna C. Escher, Virginia Ginocchio, Raffaele Iorio, Norman Junge, Ulrich Baumann, Yan Zhu, Figen Özçay, Fakher
Publié dans: HEPATOLOGY, Numéro V68IS1, Oct 2018, 2018, Page(s) 722A
Éditeur: John Wiley & Sons, Inc.

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