AAV3B Reference-2.8kb

The AAV3B reference-2.8kb material contains a 2.8kb AAV-CMV-SV40I-EGFP-WPRE-hGHpolyA genome in AAV3B capsid, with a genome titer (EGFP targeting) of 1X10^13 VG/ml. This reference is produced using the AAVone system with the pAAVone-AAV3B-CMV-EGFP-2.8kb plasmid. AAV3B is a serotype of adeno-associated virus that has shown promising potential for liver-targeted gene therapy applications. Compared to other AAV serotypes, AAV3B has a unique tissue tropism, with higher transduction efficiency in human hepatocytes. This makes it an attractive option for treating liver-specific genetic diseases such as hemophilia or metabolic disorders.

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Background

AAV references are crucial for qPCR-based vector genome quantification and fundamental for ensuring accuracy in viral vector research and gene therapy development. Our AAV reference materials undergo extensive characterization, including full ratio (mass photometry), genome titer (ddPCR), capsid titer (ELISA), purity (SDS-PAGE silver staining), and tests for bioburden, endotoxin, and mycoplasma. Quantification is based on reference material from ATCC. These thoroughly analyzed particles provide precise genome titers, well-defined full-to-empty capsid ratios, and accurate capsid titers. This comprehensive characterization is essential for standardizing measurements, ensuring consistency across experiments, and delivering reliable data for gene therapy applications and other research involving AAV vectors.
AAV references (Truly-Empty) are produced by transfection of a single mini-pHelper-AAV plasmid with the corresponding serotypes. AAV references (Full) and AAV references (Byproduct-Empty) are generated using the AAVone system, with the corresponding pAAVone plasmids carrying AAV-CMV-SV40I-EGFP-WRRE-hGHpolyA genomes.