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

Risultati finali

Public project website
CureCN audio-visual material

CureCN satellite symposium
Comparison of current treatment modalities and outcomes
Quality of life of CN patients
Natural course of CN syndrome

Pubblicazioni

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

Autori: 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
Pubblicato in: Scientific Reports, Numero 10/1, 2020, ISSN 2045-2322
Editore: 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

Autori: 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
Pubblicato in: Molecular Therapy - Methods & Clinical Development, Numero 18, 2020, Pagina/e 250-258, ISSN 2329-0501
Editore: Elsevier
DOI: 10.1016/j.omtm.2020.06.002

Diagnosis and management of secondary causes of steatohepatitis

Autori: R. Liebe; Irene Esposito; Hans H. Bock; Stephan vom Dahl; Jan Stindt; Ulrich Baumann; Ulrich Baumann; Tom Luedde; Verena Keitel
Pubblicato in: Journal of Hepatology, Numero 74(6), 2021, Pagina/e 1455-1471, ISSN 1600-0641
Editore: Elsevier
DOI: 10.1016/j.jhep.2021.01.045

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

Autori: Robert De Knegt
Pubblicato in: Liver International, Numero 14783223, 2022, Pagina/e 1593-1604, ISSN 1478-3223
Editore: Blackwell Publishing Inc.
DOI: 10.1111/liv.15239

Evading and overcoming AAV neutralization in gene therapy

Autori: Joseph Earley, Elena Piletska, Giuseppe Ronzitti, Sergey Piletsky
Pubblicato in: Trends in Biotechnology, Numero Volume 41, Numero 6, 2023, Pagina/e 836-845, ISSN 0167-7799
Editore: 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

Autori: Elena Piletska, Philippe Veron, Bérangère Bertin, Federico Mingozzi, Donald Jones, Rachel L. Norman, Joseph Earley, Kal Karim, Alvaro Garcia-Cruz, Sergey Piletsky
Pubblicato in: Nanomedicine: Nanotechnology, Biology, and Medicine, Numero Volume 52, August 2023, 2023, Pagina/e 1-10, ISSN 2322-5904
Editore: 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

Autori: 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
Pubblicato in: Molecular Therapy - Methods & Clinical Development, Numero 12, 2019, Pagina/e 157-174, ISSN 2329-0501
Editore: Elsevier
DOI: 10.1016/j.omtm.2018.12.011

Gene Therapy in Patients with the Crigler–Najjar Syndrome

Autori: 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
Pubblicato in: https://hal.science/hal-04324294, Numero 389, 2023, Pagina/e 620-631, ISSN 1533-4406
Editore: 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

Autori: 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
Pubblicato in: Human Gene Therapy, Numero 30/10, 2019, Pagina/e 1297-1305, ISSN 1043-0342
Editore: Mary Ann Liebert Inc.
DOI: 10.1089/hum.2019.143

Quality of Life in patients with Crigler-Najjar Syndrome

Autori: Junge, Aronson, Baumann, Bosma, D´Antiga, Di Giorgio, Taibi, Labrune
Pubblicato in: ESPGHAN Concgress 2024, 2024
Editore: ESPGHAN Concgress 2024

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

Autori: 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
Pubblicato in: HEPATOLOGY, Numero V68IS1, Oct 2018, 2018, Pagina/e 722A
Editore: John Wiley & Sons, Inc.

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