Prevalence and Relevance of Pre-Existing Anti-Adeno-Associated Virus Immunity in the Context of Gene Therapy for Crigler-Najjar Syndrome
- Aronson, S.J. (Sem J.)
- Veron, P. (Philippe)
- Collaud, F. (Fanny)
- Hubert, A. (Aurélie)
- Delahais, V. (Virginie)
- Honnet, G. (Géraldine)
- Knegt, R.J. (Robert) de
- Junge, N. (Norman)
- Baumann, U. (Ulrich)
- Di Giorgio, A. (Angelo)
- D'Antiga, L. (Lorenzo)
- Ginocchio, V.M. (Virginia M.)
- Brunetti-Pierri, N. (Nicola)
- Labrune, P. (Philippe)
- Beuers, U. (Ulrich)
- Bosma, P.J. (Piter)
- Mingozzi, F. (Federico)
DOIAbstract
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 (<1:5) on liver gene transfer effic
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