AAV5 Reference-4.3kb

The AAV5 reference-4.3kb material contains a 4.3kb AAV-CMV-SV40I-EGFP-WPRE-hGHpolyA genome (with a 1.5kb LacZ) in AAV5 capsid, with a genome titer (EGFP targeting) of 1X10^13 VG/ml. AAV5 is a serotype of adeno-associated virus that was originally isolated from rhesus monkeys, with unique tissue tropism and characteristics that make it useful in specific gene therapy applications. AAV5 has demonstrated an ability to transduce various tissues, including the brain, lungs, and liver. Gene therapy based on AAV5 has been approved for human use, such as Roctavian (valoctocogene roxaparvovec), developed by BioMarin Pharmaceutical for the treatment of hemophilia A.

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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.