ArticleMolecular therapy. Methods & clinical development2023
Adeno-associated virus serotype 9 antibody seroprevalence for patients in the United States with spinal muscular atrophy.
Article in Molecular therapy. Methods & clinical development, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers, 1 of them a synthesis that pooled it.
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The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
10 citing papers in PubMed, 1 synthesis or guideline pooled it, 10 citations in OpenAlex.
- Systematic review for economic evaluations on newborn screening for spinal muscular atrophy.Journal of neuromuscular diseases · 2025Pooled it
- Onasemnogene abeparvovec gene therapy for treatment of patients with spinal muscular atrophy: Updated real-world practical considerations.Journal of neuromuscular diseases · 2026Review
- Environmental and developmental factors shape anti-AAV immunity in pigs.Gene therapy · 2026Article
- Foods for special medical purposes for the dietary therapy of rare diseases: Current status and future prospects.Intractable & rare diseases research · 2026Review
- Reduction of Preexisting AAV9 Antibody Titers Before Onasemnogene Abeparvovec Administration in Twins With Spinal Muscular Atrophy.Neurology · 2025Article
- A cost-utility analysis of newborn screening for spinal muscular atrophy in Canada.Orphanet journal of rare diseases · 2025Article
- Structural characterization of antibody-responses following Zolgensma treatment for AAV capsid engineering to expand patient cohorts.Nature communications · 2025Article
- Adeno-associated virus serotype 9 antibodies in neonates and young children: Seroprevalence and kinetics.Molecular therapy. Methods & clinical development · 2024Article
- Thrombotic Microangiopathy as an Emerging Complication of Viral Vector-Based Gene Therapy.Kidney international reports · 2024Review
- Preexisting antibody assays for gene therapy: Considerations on patient selection cutoffs and companion diagnostic requirements.Molecular therapy. Methods & clinical development · 2024Review
Corrections and comments
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Authors and funding
6 authors at 6 institutions in 3 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Onasemnogene abeparvovec is a recombinant adeno-associated virus serotype 9 (AAV9) vector-based gene therapy for spinal muscular atrophy (SMA). Patients with elevated titers of anti-AAV9 antibodies (AAV9-Ab) should not receive onasemnogene abeparvovec because of potential safety and efficacy implications. We conducted a retrospective study to describe the seroprevalence of anti-AAV9 binding antibodies for pediatric patients with SMA in the United States. At initial testing, 13.0% (115 of 882) of patients (mean [SD] age, 26.29 [33.66] weeks) had elevated AAV9-Ab titers. The prevalence of elevated titers decreased as age increased, with 18.2% (92 of 507) of patients ≤3 months old but only 1.1% (1 of 92) of patients ≥21 months old having elevated titers. This suggests transplacental maternal transfer of antibodies. No patterns of geographic variations in AAV9-Ab prevalence were confirmed. Elevated AAV9-Ab titers in children <6 weeks old decreased in all circumstances. Lower magnitudes of elevated titers declined more rapidly than greater magnitudes. Retesting was completed at the discretion of the treating clinician, so age at testing and time between tests varied. AAV9-Ab retesting should be considered when patients have elevated titers, and elevations at a young age are not a deterrent to eventual onasemnogene abeparvovec administration. Early disease-modifying treatment for SMA leads to optimal outcomes.
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Registered trials
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