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

Resultado final

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

Publicaciones

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

Autores: 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
Publicado en: Scientific Reports, Edición 10/1, 2020, ISSN 2045-2322
Editor: 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

Autores: 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
Publicado en: Molecular Therapy - Methods & Clinical Development, Edición 18, 2020, Página(s) 250-258, ISSN 2329-0501
Editor: Elsevier
DOI: 10.1016/j.omtm.2020.06.002

Diagnosis and management of secondary causes of steatohepatitis

Autores: R. Liebe; Irene Esposito; Hans H. Bock; Stephan vom Dahl; Jan Stindt; Ulrich Baumann; Ulrich Baumann; Tom Luedde; Verena Keitel
Publicado en: Journal of Hepatology, Edición 74(6), 2021, Página(s) 1455-1471, ISSN 1600-0641
Editor: Elsevier
DOI: 10.1016/j.jhep.2021.01.045

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

Autores: Robert De Knegt
Publicado en: Liver International, Edición 14783223, 2022, Página(s) 1593-1604, ISSN 1478-3223
Editor: Blackwell Publishing Inc.
DOI: 10.1111/liv.15239

Evading and overcoming AAV neutralization in gene therapy

Autores: Joseph Earley, Elena Piletska, Giuseppe Ronzitti, Sergey Piletsky
Publicado en: Trends in Biotechnology, Edición Volume 41, Edición 6, 2023, Página(s) 836-845, ISSN 0167-7799
Editor: 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

Autores: Elena Piletska, Philippe Veron, Bérangère Bertin, Federico Mingozzi, Donald Jones, Rachel L. Norman, Joseph Earley, Kal Karim, Alvaro Garcia-Cruz, Sergey Piletsky
Publicado en: Nanomedicine: Nanotechnology, Biology, and Medicine, Edición Volume 52, August 2023, 2023, Página(s) 1-10, ISSN 2322-5904
Editor: 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

Autores: 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
Publicado en: Molecular Therapy - Methods & Clinical Development, Edición 12, 2019, Página(s) 157-174, ISSN 2329-0501
Editor: Elsevier
DOI: 10.1016/j.omtm.2018.12.011

Gene Therapy in Patients with the Crigler–Najjar Syndrome

Autores: 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
Publicado en: https://hal.science/hal-04324294, Edición 389, 2023, Página(s) 620-631, ISSN 1533-4406
Editor: 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

Autores: 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
Publicado en: Human Gene Therapy, Edición 30/10, 2019, Página(s) 1297-1305, ISSN 1043-0342
Editor: Mary Ann Liebert Inc.
DOI: 10.1089/hum.2019.143

Quality of Life in patients with Crigler-Najjar Syndrome

Autores: Junge, Aronson, Baumann, Bosma, D´Antiga, Di Giorgio, Taibi, Labrune
Publicado en: ESPGHAN Concgress 2024, 2024
Editor: ESPGHAN Concgress 2024

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

Autores: 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
Publicado en: HEPATOLOGY, Edición V68IS1, Oct 2018, 2018, Página(s) 722A
Editor: John Wiley & Sons, Inc.

Resultado final

Publicaciones

Compartir esta página

Descargar