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

Publikováno v: 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⟩

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,

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::5c1a2d9c9f9b11cded8a89710f8f8580
https://pubmed.ncbi.nlm.nih.gov/31502485