Description
Byproduct-empty AAV particles are unintentional outcomes that can occur during the production of regular AAV vectors. Byproduct-empty AAV particles are not truly devoid of genetic material, as they might contain partial or incomplete genomes. They serve as valuable tools for various stages of gene therapy development and characterization.
- Reference Materials: Well-characterized empty AAV capsids, can serve as standardized reference materials for analytical assays.
- Quality Control: During the manufacturing process of AAV vectors, using well-characterized capsids as reference materials allows manufacturers to monitor and ensure the quality, purity, and consistency of their AAV products.
- Biodistribution Studies: Empty AAV can be used in biodistribution studies to assess how different capsids distribute in various tissues after administration.
- Immunogenicity Assessment: By exposing the immune system to well-characterized AAV capsids, researchers can evaluate the immune responses induced by specific serotypes..
- Comparative Studies: Empty AAV allow researchers to compare the characteristics of different AAV serotypes without the confounding effects of transgene expression.
- Process Development: Empty AAV can be used in the development and optimization of manufacturing processes for AAV vectors.
- Assay Development: Empty AAV capsids serve as positive controls in various assays, aiding the development of new methods for AAV characterization, stability testing, and potency determination.
- Gene Therapy Capsid Decoys: The presence of an excess of empty AAV capsids may effectively absorb low-level neutralizing antibodies (NAbs) and non-NAbs, permitting transduction even in their presence.