National Institutes of Health/National Institute of General Medical Sciences (NIH/NIGMS)
NIH GM082946
米国
引用
ジャーナル: J Virol / 年: 2023 タイトル: Structural and antigenic characterization of the avian adeno-associated virus capsid. 著者: Jane Hsi / Mario Mietzsch / Paul Chipman / Sandra Afione / Allison Zeher / Rick Huang / John Chiorini / Robert McKenna / 要旨: 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 ...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.