ArticleMolecular therapy. Methods & clinical development2024
An investigation of the immune epitopes of adeno-associated virus capsid-derived peptides among hemophilia patients.
Article in Molecular therapy. Methods & clinical development, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.
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2 citing papers in PubMed.
- Sequence-based artificial intelligence-guided inhibitory peptides to counteract AAV neutralizing antibodies in gene therapy.Molecular therapy. Advances · 2026Article
- Efficient Scaling up EV-AAVs Production via Cellular Nanoporation for Familial Hypercholesterolaemia Therapy.Journal of extracellular vesicles · 2025Article
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12 authors.
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Abstract
Adeno-associated virus (AAV) is an optimal gene vector for monogenic disorders. However, neutralizing antibodies (Nabs) against AAV hinder its widespread application in gene therapy. In this study, we biosynthesized peptides recognized by the binding antibodies (Babs) from the sera containing high Nab titers against AAV2. We established four immunological methods to detect immune epitopes of the AAV2-derived peptides, including a Bab assay, Nab assay, B cell receptor (BCR) detecting assay, and immunoglobin-producing B cell enzyme-linked immunosorbent spot (B cell ELISpot) assay. Correlations among the epitopes determined by these four methods were analyzed using the serum samples and peripheral blood mononuclear cells (PBMC) from 89 patients with hemophilia A/B. As decoys, the peptides' ability to block the Nab of AAV2 particles was assessed using AAV transduction models both
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