ArticleHuman gene therapy2024
Prevalence of Neutralizing Antibodies Against AAV Serotypes 2 and 9 in Healthy Participants from Multiple Centers Across China and Patients with DMD/BMD.
Article in Human gene therapy, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.
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Who cites it
7 citing papers in PubMed.
- Seroprevalence of anti-AAV neutralizing antibodiesin healthy and retinitis pigmentosa cohorts: A multi-province study in China.Molecular therapy. Advances · 2026Article
- Environmental and developmental factors shape anti-AAV immunity in pigs.Gene therapy · 2026Article
- Adeno-Associated Virus Toxicity in Duchenne Muscular Dystrophy: Mechanisms and Clinical Considerations.Genes · 2026Review
- Global seroprevalence of neutralizing and total antibodies against AAV vectors and their impact on the clinical gene therapy landscape: a scoping review.Frontiers in medicine · 2026Review
- Preclinical evaluation of AL-001, a gene therapy for wet age-related macular degeneration.Frontiers in genetics · 2026Article
- AAV microdystrophin gene replacement therapy for Duchenne muscular dystrophy: progress and prospects.Gene therapy · 2025Review
- Prevalence of Neutralizing Antibodies to AAV2 and AAV9 in Individuals with Niemann-Pick Disease, Type C1.Human gene therapy · 2025Article
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Authors and funding
10 authors.
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Abstract
To facilitate adeno-associated virus (AAV)-mediated gene therapy in China, we conducted a study on the distribution of AAV-neutralizing antibodies (NAbs) in healthy subjects and in patients with Duchenne muscular dystrophy (DMD)/Becker muscular dystrophy (BMD). A total of 352 healthy adult controls (ACs) from a national multicenter study, 100 schoolchild controls (SCs), and 281 patients with DMD/BMD from Peking Union Medical College Hospital were enrolled in this study. Cell-based inhibition assays were applied, and serum samples demonstrating 50% inhibition of infection were considered positive. The seroprevalence of AAV2 and AAV9 NAbs among the 733 participants was 86.1% and 56.3%, respectively. The AAV2 NAbs and AAV9 NAbs positivity rates in the AC, SC, and DMD/BMD groups were 97.4%/86.6%, 100.0%/17.0%, and 66.9%/32.4%, respectively. The seroprevalence of AAV NAbs gradually increased with age, especially in AAV9 NAbs. Females tended to have higher positivity rate than males. Over 85% of ACs had overlapping AAV9 and AAV2 infection. However, being positive for only AAV2 NAbs in the SC group was common, and 30.6% of patients with DMD/BMD were negative for both AAV2 and AAV9 NAbs. Our findings reveal that a significant proportion of patients with DMD/BMD were negative for AAV2 and AAV9 NAbs, which is the population that is most amenable to being treated with gene therapy.
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