Ontology highlight
ABSTRACT: Importance
AAVs are extensively studied as promising therapeutic gene delivery vectors. In order to circumvent pre-existing antibodies targeting primate-based AAV capsids, the AAAV capsid was evaluated as an alternative to primate-based therapeutic vectors. Despite the high sequence diversity, the AAAV capsid was found to bind to a common glycan receptor, terminal galactose, which is also utilized by other AAVs already being utilized in gene therapy trials. However, contrary to the initial hypothesis, AAAV was recognized by approximately 30% of human sera tested. Structural and sequence comparisons point to conserved epitopes in the fivefold region of the capsid as the reason determinant for the observed cross-reactivity.
SUBMITTER: Hsi J
PROVIDER: S-EPMC10617571 | biostudies-literature | 2023 Oct
REPOSITORIES: biostudies-literature
Hsi Jane J Mietzsch Mario M Chipman Paul P Afione Sandra S Zeher Allison A Huang Rick R Chiorini John J McKenna Robert R
Journal of virology 20230913 10
<h4>Importance</h4>AAVs are extensively studied as promising therapeutic gene delivery vectors. In order to circumvent pre-existing antibodies targeting primate-based AAV capsids, the AAAV capsid was evaluated as an alternative to primate-based therapeutic vectors. Despite the high sequence diversity, the AAAV capsid was found to bind to a common glycan receptor, terminal galactose, which is also utilized by other AAVs already being utilized in gene therapy trials. However, contrary to the initi ...[more]