Prevalence and Relevance of Pre-Existing Anti-Adeno-Associated Virus Immunity in the Context of Gene Therapy for Crigler–Najjar Syndrome

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...

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Bibliografiske detaljer
Udgivet i:Hum Gene Ther
Main Authors: Aronson, Sem J., Veron, Philippe, Collaud, Fanny, Hubert, Aurélie, Delahais, Virginie, Honnet, Géraldine, de Knegt, Robert J., Junge, Norman, Baumann, Ulrich, Di Giorgio, Angelo, D'Antiga, Lorenzo, Ginocchio, Virginia M., Brunetti-Pierri, Nicola, Labrune, Philippe, Beuers, Ulrich, Bosma, Piter J., Mingozzi, Federico
Format: Artigo
Sprog:Inglês
Udgivet: Mary Ann Liebert, Inc., publishers 2019
Fag:
Research Articles
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6763963/
https://ncbi.nlm.nih.gov/pubmed/31502485
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2019.143
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