Truly empty capsids devoid of any genomic materials. 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.