Prevalence and Relevance of Pre-Existing Anti-Adeno-Associated Virus Immunity in the Context of Gene Therapy for Crigler-Najjar Syndrome
| Autor: | Sem J. Aronson, Angelo Di Giorgio, Ulrich Baumann, Virginie Delahais, Lorenzo D'Antiga, Fanny Collaud, Aurélie Hubert, Géraldine Honnet, Federico Mingozzi, Nicola Brunetti-Pierri, Philippe Labrune, Norman Junge, Robert J. de Knegt, Ulrich Beuers, Virginia Maria Ginocchio, Piter J. Bosma, Philippe Veron |
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| Přispěvatelé: | Approches génétiques intégrées et nouvelles thérapies pour les maladies rares (INTEGRARE), École pratique des hautes études (EPHE), Université Paris sciences et lettres (PSL)-Université Paris sciences et lettres (PSL)-Université d'Évry-Val-d'Essonne (UEVE)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Généthon, AP-HP - Hôpital Antoine Béclère [Clamart], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP), Gastroenterology and Hepatology, Erasmus University Medical Center [Rotterdam] (Erasmus MC), Università degli studi di Napoli Federico II, University of Amsterdam [Amsterdam] (UvA), Graduate School, Tytgat Institute for Liver and Intestinal Research, AGEM - Digestive immunity, AGEM - Endocrinology, metabolism and nutrition, AGEM - Inborn errors of metabolism, École Pratique des Hautes Études (EPHE), University of Naples Federico II = Università degli studi di Napoli Federico II, Gastroenterology & Hepatology, Aronson, S. J., Veron, P., Collaud, F., Hubert, A., Delahais, V., Honnet, G., De Knegt, R. J., Junge, N., Baumann, U., Di Giorgio, A., D'Antiga, L., Ginocchio, virginia maria, Brunetti-Pierri, N., Labrune, P., Beuers, U., Bosma, P. J., Mingozzi, F., Laboratoire Traitement du Signal et de l'Image (LTSI), Université de Rennes 1 (UR1), Université de Rennes (UNIV-RENNES)-Université de Rennes (UNIV-RENNES)-Institut National de la Santé et de la Recherche Médicale (INSERM), École pratique des hautes études (EPHE)-Université d'Évry-Val-d'Essonne (UEVE)-GENETHON 3-Institut National de la Santé et de la Recherche Médicale (INSERM), COMUE Université Côte d'Azur (2015 - 2019) (COMUE UCA) |
| Rok vydání: | 2019 |
| Předmět: |
Serotype
Male anti-AAV neutralizing antibodie Crigler–Najjar syndrome [SDV]Life Sciences [q-bio] Genetic enhancement anti-AAV neutralizing antibodies Gene Expression medicine.disease_cause Antibodies Viral [SDV.IMM.II]Life Sciences [q-bio]/Immunology/Innate immunity Mice 0302 clinical medicine Glucuronosyltransferase Child Adeno-associated virus Research Articles Crigler-Najjar Syndrome 0303 health sciences biology [SDV.MHEP.EM]Life Sciences [q-bio]/Human health and pathology/Endocrinology and metabolism Dependovirus 3. Good health Titer unconjugated hyperbilirubinemia [SDV.IMM.IA]Life Sciences [q-bio]/Immunology/Adaptive immunology Liver 030220 oncology & carcinogenesis Child Preschool Phenobarbital Molecular Medicine Female Antibody AAV gene therapy Plasmids Adult Adolescent pre-existing immunity Transfection Virus 03 medical and health sciences Capsid Immunity Genetics medicine Animals Humans Molecular Biology 030304 developmental biology business.industry Immunization Passive Bilirubin Genetic Therapy Phototherapy medicine.disease Antibodies Neutralizing Immunity Innate Mice Inbred C57BL HEK293 Cells Immunology biology.protein UGT1A1 business Excitatory Amino Acid Antagonists |
| Zdroj: | Human Gene Therapy Human Gene Therapy, Mary Ann Liebert, 2019, 30, pp.1297-1305. ⟨10.1089/hum.2019.143⟩ Human gene therapy, 30(10), 1297-1305. Mary Ann Liebert Inc. Human Gene Therapy, 2019, 30, pp.1297-1305. ⟨10.1089/hum.2019.143⟩ Human Gene Therapy, 30(10), 1297-1305. Mary Ann Liebert Inc. Hum Gene Ther Hum Gene Ther, 2019, 30, pp.1297-1305. ⟨10.1089/hum.2019.143⟩ |
| ISSN: | 1557-7422 1043-0342 |
| DOI: | 10.1089/hum.2019.143⟩ |
| Popis: | International audience; Adeno-associated virus (AAV) vector-mediated gene therapy is currently evaluated as a potential treatment for Crigler-Najjar syndrome (CN) (NCT03466463). Pre-existing immunity to AAV is known to hinder gene transfer efficacy, restricting enrollment of seropositive subjects in ongoing clinical trials. We assessed the prevalence of anti-AAV serotype 8 (AAV8) neutralizing antibodies (NAbs) in subjects affected by CN and investigated the impact of low NAb titers ( |
| Databáze: | OpenAIRE |
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