ArticleMolecular therapy. Methods & clinical development2023
In-depth comparison of Anc80L65 and AAV9 retinal targeting and characterization of cross-reactivity to multiple AAV serotypes in humans.
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 6 papers, 1 of them a synthesis that pooled it.
What it found
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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
6 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Retinal Viral Gene Therapy: Impact of Route of Administration on Serious Adverse Events-A Systematic Review.Clinical & experimental ophthalmology · 2025Pooled it
- Adeno-Associated Virus Vector Mediated Gene Therapy: A Promising Approach to Transform Hypertrophic Cardiomyopathy Treatment.Biotechnology journal · 2026Review
- Screening of Single-Domain Antibodies to Adeno-Associated Viruses with Cross-Serotype Specificity and a Wide pH Tolerance.Viruses · 2025Article
- Recent Advances in Designing Adeno-Associated Virus-Based Vaccines Against Viral Infections.Pharmaceutics · 2024Review
- Efficient AAV9 Purification Using a Single-Step AAV9 Magnetic Affinity Beads Isolation.International journal of molecular sciences · 2024Article
- Adeno-associated virus vectors for retinal gene therapy in basic research and clinical studies.Frontiers in medicine · 2023Review
Corrections and comments
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Authors and funding
9 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Anc80L65 is a synthetic, ancestral adeno-associated virus that has high tropism toward retinal photoreceptors after subretinal injection in mice and non-human primates. We characterized, for the first time, the post-intravitreal cell-specific transduction profile of Anc80L65 compared with AAV9. Here we use Anc80L65 and AAV9 to intravitreally deliver a copy of the gene encoding GFP into WT C57Bl/6J mice. GFP expression was driven by one of two clinically relevant promoters, chicken β actin (CB) or truncated MECP2 (P546). After qualitative assessment of relative GFP expression, we found Anc80L65 and AAV9 to have similar transduction profiles. Through the development of a novel method for quantifying GFP-positive retinal cells, we found Anc80L65 to have higher tropism in Müller glia and AAV9 to have higher tropism in horizontal cells. In addition, we found P546 to promote GFP expression at a more moderate level compared with the high levels seen under the CB promoter. Finally, for the first time, we characterized Anc80L65 cross-reactivity in human sera; 83% of patients with AAV2 pre-existing antibodies were found to be seropositive for Anc80L65. This study demonstrates the expanded therapeutic applications of Anc80L65 to treat retinal disease and provides the first insights to Anc80L65 pre-existing immunity in humans.
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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.