ArticleMolecular therapy : the journal of the American Society of Gene Therapy2012
Neutralizing antibodies against AAV serotypes 1, 2, 6, and 9 in sera of commonly used animal models.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 109 papers.
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Who cites it
109 citing papers in PubMed, 168 citations in OpenAlex.
- Retinal AAV8-RS1 Gene Therapy for X-Linked Retinoschisis: Initial Findings from a Phase I/IIa Trial by Intravitreal Delivery.Molecular therapy : the journal of the American Society of Gene Therapy · 2018Trial
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- Murine toxicology assessment of avgn7.2, a novel gene therapeutic for inclusion body myositis and other muscle wasting diseases.Gene therapy · 2026Article
- Current regulatory requirements for assessment of immunogenicity for gene therapy medicinal products.Cell reports. Medicine · 2025Review
- Fiber Photometry Analysis of Spontaneous Dopamine Signals: TheACS chemical neuroscience · 2025Article
- Lessons Learned from Monitoring Adeno-Associated Virus-9 Neutralizing Antibody (AAV9-NAb) Seroconversion in a Cohort of Cynomolgus Macaques (Macaca fascicularis).Journal of the American Association for Laboratory Animal Science : JAALAS · 2025Article
- Fiber photometry analysis of spontaneous dopamine signals: The z-scored data are not the data.bioRxiv : the preprint server for biology · 2025Article
- Challenges in Humoral Immune Response to Adeno-Associated Viruses Determination.International journal of molecular sciences · 2025Review
- Article
- AAV Capsid Screening for Translational Pig Research Using a Mouse Xenograft Liver Model.bioRxiv : the preprint server for biology · 2024Article
- Challenges and prospects in geriatric epilepsy treatment: the role of the blood-brain barrier in pharmacotherapy and drug delivery.Frontiers in aging neuroscience · 2024Review
- Understanding AAV vector immunogenicity: from particle to patient.Theranostics · 2024Review
- Messenger RNA-encoded antibody approach for targeting extracellular and intracellular tau.Brain communications · 2024Article
- Gene therapy for the heart: encapsulated viruses to the rescue.Extracellular vesicles and circulating nucleic acids · 2024Article
- AAV Vectors Pseudotyped with Capsids from Porcine and Bovine Species Mediate In Vitro and In Vivo Gene Delivery.Viruses · 2023Article
- Drug Delivery to the Brain: Recent Advances and Unmet Challenges.Pharmaceutics · 2023Review
- Structure-guided AAV capsid evolution strategies for enhanced CNS gene delivery.Nature protocols · 2023Review
- Chemogenetic Silencing of NaeNeuro · 2023Article
- Regenerative medicine improve neurodegenerative diseases.Cell and tissue banking · 2023Review
49 more citing papers are in PubMed but not listed here.
Corrections and comments
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Authors and funding
8 authors at 2 institutions in 1 country.
Funding
Abstract
Adeno-associated virus (AAV)-based vectors are promising gene delivery vehicles for human gene transfer. One significant obstacle to AAV-based gene therapy is the high prevalence of neutralizing antibodies in humans. Until now, it was thought that, except for nonhuman primates, pre-existing neutralizing antibodies are not a problem in small or large animal models for gene therapy. Here, we demonstrate that sera of several animal models of cardiovascular diseases harbor pre-existing antibodies against the cardiotropic AAV serotypes AAV1, AAV6, and AAV9 and against AAV2. The neutralizing antibody titers vary widely both between species and between serotypes. Of all species tested, rats displayed the lowest levels of neutralizing antibodies. Surprisingly, naive mice obtained directly from commercial vendors harbored neutralizing antibodies. Of the large animal models tested, the neutralization of AAV6 transduction by dog sera was especially pronounced. Sera of sheep and rabbits showed modest neutralization of AAV transduction whereas porcine sera strongly inhibited transduction by all AAV serotypes and displayed the largest variation between individual animals. Importantly, neutralizing antibody titers as low as 1/4 completely prevented in vivo transduction by AAV9 in rats. Our results suggest that prescreening of animals for neutralizing antibodies will be important for future gene transfer experiments in these animal models.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.