Pre-existing Anti-AAV9 antibodies in the Chinese healthy and rare disease populations: Implications for gene therapy.

ABSTRACT: The adeno-associated virus 9 (AAV9) vector was particularly notable for its broad tissue tropism, making it a preferred vector for gene therapy. Goals: The study aimed to investigate the patterns of pre-existing immunity against AAV9 in the Chinese population. In this study, we conducted a serological research from November 2022 to June 2024. The study included 341 participants in total with age ranged from 0 to 90 years old: 270 healthy individuals, 30 pediatric patients and 41 adults with rare diseases. Total AAV9-binding antibodies (TAbs) and neutralizing antibodies (NAbs) were measured. The seroprevalence of anti-AAV9 NAbs showed no significant differences between healthy individuals and rare disease patients across both pediatric and adult groups. Newborns exhibited a high NAb-positiv

SUBMITTER: Zhao Q 

PROVIDER: S-EPMC11925575 | biostudies-literature | 2025 Apr

REPOSITORIES: biostudies-literature