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

Project Information

CureCN

Grant agreement ID: 755225

DOI 10.3030/755225

Project closed

EC signature date 5 December 2017

Start date 1 January 2018

End date 30 June 2023

Funded under

• SOCIETAL CHALLENGES - Health, demographic change and well-being
• Treating and managing disease

Total cost € 6 249 103,75

EU contribution € 6 249 103,75

Coordinated by

ASSOCIATION GENETHON
France

Deliverables

Websites, patent fillings, videos etc. (2)

Public project website
CureCN audio-visual material

Documents, reports (4)

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

Publications

Peer reviewed articles (9)

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

Author(s): 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
Published in: Scientific Reports, Issue 10/1, 2020, ISSN 2045-2322
Publisher: 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

Author(s): 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
Published in: Molecular Therapy - Methods & Clinical Development, Issue 18, 2020, Page(s) 250-258, ISSN 2329-0501
Publisher: Elsevier
DOI: 10.1016/j.omtm.2020.06.002

Diagnosis and management of secondary causes of steatohepatitis

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

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

Author(s): Robert De Knegt
Published in: Liver International, Issue 14783223, 2022, Page(s) 1593-1604, ISSN 1478-3223
Publisher: Blackwell Publishing Inc.
DOI: 10.1111/liv.15239

Evading and overcoming AAV neutralization in gene therapy

Author(s): Joseph Earley, Elena Piletska, Giuseppe Ronzitti, Sergey Piletsky
Published in: Trends in Biotechnology, Issue Volume 41, Issue 6, 2023, Page(s) 836-845, ISSN 0167-7799
Publisher: 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

Author(s): Elena Piletska, Philippe Veron, Bérangère Bertin, Federico Mingozzi, Donald Jones, Rachel L. Norman, Joseph Earley, Kal Karim, Alvaro Garcia-Cruz, Sergey Piletsky
Published in: Nanomedicine: Nanotechnology, Biology, and Medicine, Issue Volume 52, August 2023, 2023, Page(s) 1-10, ISSN 2322-5904
Publisher: 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

Author(s): 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
Published in: Molecular Therapy - Methods & Clinical Development, Issue 12, 2019, Page(s) 157-174, ISSN 2329-0501
Publisher: Elsevier
DOI: 10.1016/j.omtm.2018.12.011

Gene Therapy in Patients with the Crigler–Najjar Syndrome

Author(s): 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
Published in: https://hal.science/hal-04324294, Issue 389, 2023, Page(s) 620-631, ISSN 1533-4406
Publisher: 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

Author(s): 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
Published in: Human Gene Therapy, Issue 30/10, 2019, Page(s) 1297-1305, ISSN 1043-0342
Publisher: Mary Ann Liebert Inc.
DOI: 10.1089/hum.2019.143

Conference proceedings (2)

Quality of Life in patients with Crigler-Najjar Syndrome

Author(s): Junge, Aronson, Baumann, Bosma, D´Antiga, Di Giorgio, Taibi, Labrune
Published in: ESPGHAN Concgress 2024, 2024
Publisher: ESPGHAN Concgress 2024

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

Author(s): 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
Published in: HEPATOLOGY, Issue V68IS1, Oct 2018, 2018, Page(s) 722A
Publisher: John Wiley & Sons, Inc.