Empty AAV particles are indeed versatile tools with various applications in AAV-based gene therapy development, quality control, and basic research.
There is two different types of empty particles:
Truly-Empty AAV Particles
Truly-empty AAV particles are intentionally created through a specialized production process where only the necessary components for AAV replication (e.g., ad helper and AAV helper) are provided. Truly-empty AAV particles do not contain any genetic material, making them entirely empty capsids. They lack the therapeutic transgene that is typically present in AAV vectors.
Byproduct-Empty AAV Particles
Byproduct-empty AAV particles are unintentional outcomes that can arise during the production of regular AAV vectors. They are not deliberately generated but are a result of the production process. Unlike truly-empty AAV particles, byproduct-empty AAV particles may contain partial or incomplete genomes. They are not completely devoid of genetic material, and this genetic content can vary in terms of completeness. Byproduct-empty AAV particles are not the primary focus of production and can sometimes be considered impurities or contaminants in the final AAV vector preparation.
The key distinction between these two types of empty particles lies in their intentional generation and genetic content. Truly-empty AAV particles are purposely created without genetic material and are used as a starting point for constructing therapeutic AAV vectors. In contrast, byproduct-empty AAV particles are unintentional and may contain some genetic material, potentially affecting the purity and function of the final vector.
Researchers and manufacturers need to be aware of this distinction and carefully control the production process to minimize the presence of byproduct-empty AAV particles, as their unintended genetic content could impact the safety and efficacy of gene therapy applications.
| Truly-empty AAV particles | AAV1, AAV2, AAV3B, AAV5, AAV6, AAV7, AAV8, AAV9, AAV10, AAV11, AAV12, AAV13, AAVDJ, AAVrh.10, AAVrh.74, AAV2-retro |
| Byproduct-Empty AAV particles | Variety empty AAV serotypes from AAV-CAG-EGFP preparation or AAV-CMV-EGFP preparation |
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