Preexisting Neutralizing Antibodies against Different Adeno-Associated Virus Serotypes in Humans and Large Animal Models for Gene Therapy.
AAV serotypes
Adeno-associated virus
Anti-AAV immune response
Large animal models
Neutralizing antibodies
Retinal gene therapy
Journal
Advances in experimental medicine and biology
ISSN: 0065-2598
Titre abrégé: Adv Exp Med Biol
Pays: United States
ID NLM: 0121103
Informations de publication
Date de publication:
2023
2023
Historique:
medline:
17
7
2023
pubmed:
13
7
2023
entrez:
13
7
2023
Statut:
ppublish
Résumé
Gene therapy is a potential cure for several inherited retinal dystrophies, and adeno-associated virus (AAV) has emerged as a vector of choice for therapeutic gene delivery to the retina. However, prior exposure to AAVs can cause a humoral immune response resulting in the presence of antibodies in the serum, which can subsequently interfere with the AAV-mediated gene therapy. The antibodies bind specifically to a serotype but often display broad cross-reactivity. A subset of these antibodies called neutralizing antibodies (NABs) can render the AAV inactive, thereby reducing the efficacy of the therapy. The preexisting NAB levels against different serotypes vary by species, and these variations need to be considered while designing studies. Since large animals often serve as preclinical models to test gene therapies, in this review we compile studies reporting preexisting NABs against commonly used AAV serotypes in humans and large animal models and discuss strategies to deal with NABs.
Identifiants
pubmed: 37440023
doi: 10.1007/978-3-031-27681-1_18
doi:
Substances chimiques
Antibodies, Neutralizing
0
Antibodies, Viral
0
Types de publication
Review
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
117-123Informations de copyright
© 2023. The Author(s), under exclusive license to Springer Nature Switzerland AG.
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