AAV References
AAV References
In the rapidly evolving field of gene therapy, the reliability and accuracy of research depend significantly on the use of high-quality reference materials. Both researchers and regulatory bodies like the FDA emphasize the importance of well-characterized AAV reference materials. AAVnerGene provides two types of AAV reference materials: AAV References (Full) and AAV References (Empty).
- AAV References (Full) are essential for qPCR-based vector genome quantification. These thoroughly analyzed particles offer precise genome titers, well-defined full-to-empty capsid ratios, and accurate capsid titers. This comprehensive characterization is vital for standardizing measurements, ensuring consistency across experiments, and providing reliable data for gene therapy applications and other research involving AAV vectors. All AAV References (Full) are produced using our unique and consistent AAVone production system.
- AAV References (Empty) serve as versatile tools for various applications in AAV-based gene therapy development, quality control, and basic research. There are 2 types of AAV References (Empty):
- AAV References (Truly-Empty) are purposefully created through a specialized production process that includes only the essential components for AAV replication (e.g., mini-pHelper-AAV2). These references contain no genetic material, resulting in completely empty capsids devoid of the therapeutic transgene typically found in AAV vectors. In contrast,
- AAV References (Byproduct-Empty) are unintentional byproducts of the standard AAV vector production process. They are not deliberately generated and may contain partial or incomplete genomes.
- The key distinction between these 2 types of empty particles lies in their method of generation and genetic content, which can potentially impact the purity and functionality of the final vector [Wang et al., 2016]. Researchers and manufacturers must recognize this distinction and exercise careful control over the production process to minimize the presence of byproduct-empty AAV particles. The unintended genetic content in these particles could potentially affect the safety and efficacy of gene therapy applications.
AAV References at AAVnerGene
AAV references (Full) and AAV references (Byproduct-Empty) are generated using the AAVone system, with the corresponding pAAVone plasmids carrying a 2.8 kb AAV-CMV-SV40I-EGFP-WPRE-hGHpolyA genome. AAV references (Truly-Empty) are produced by transfection of a single mini-pHelper-AAV plasmid with the corresponding serotypes. AAV reference materials are available for various serotypes, including AAV1, AAV2, AAV5, AAV6, AAV8, AAV9, AAVrh.10, and AAVrh.74. AAV references with other serotypes and genomes are available upon request.
| AAV References | Serotype | Genome | Production Plasmids |
| AAV References (Full) |
AAV1, AAV2, AAV5, AAV6, AAV8, AAV9, AAVrh.10, AAVrh.74 | Full of AAV-CMV-SV40I-EGFP-WPRE-hGHpolyA-2.8kb | pAAVone-AAVs-CMV-EGFP-2.8kb |
| AAV References (Truly-Empty) |
AAV1, AAV2, AAV5, AAV6, AAV8, AAV9, AAVrh.10, AAVrh.74 | No | mini-pHelper-AAVs |
| AAV References (Byproduct-Empty) |
AAV1, AAV2, AAV5, AAV6, AAV8, AAV9, AAVrh.10, AAVrh.74 | Partial of AAV-CMV-SV40I-EGFP-WPRE-hGHpolyA-2.8kb | pAAVone-AAVs-CMV-EGFP-2.8kb |
AAV Reference (Full)
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AAV References (Truly-Empty)
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AAV References (Byproduct-Empty)
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AAVnerGene offers high quality, short turnaround, high efficiency custom AAV packaging services with our unique single-plasmid AAV production system (AAVone), dual-plasmid AAV production system(AAVdual) or improved triple-plasmid system(AAVtri) with our mini-pHelper.
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