Prevalence and Relevance of Pre-Existing Anti-Adeno-Associated Virus Immunity in the Context of Gene Therapy for Crigler-Najjar Syndrome
| العنوان: | Prevalence and Relevance of Pre-Existing Anti-Adeno-Associated Virus Immunity in the Context of Gene Therapy for Crigler-Najjar Syndrome |
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| المؤلفون: | 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 |
| المساهمون: | 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) |
| المصدر: | 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⟩ |
| سنة النشر: | 2019 |
| مصطلحات موضوعية: | 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 |
| الوصف: | 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 ( |
| وصف الملف: | application/pdf |
| تدمد: | 1557-7422 1043-0342 |
| الوصول الحر: | https://explore.openaire.eu/search/publication?articleId=doi_dedup___::5c1a2d9c9f9b11cded8a89710f8f8580Test https://pubmed.ncbi.nlm.nih.gov/31502485Test |
| حقوق: | OPEN |
| رقم الانضمام: | edsair.doi.dedup.....5c1a2d9c9f9b11cded8a89710f8f8580 |
| قاعدة البيانات: | OpenAIRE |
| تدمد: | 15577422 10430342 |
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