AAVrh.74 Reference-4.3kb

The AAVrh.74 reference-4.3kb material contains a 4.3kb AAV-CMV-SV40I-EGFP-WPRE-hGHpolyA genome (with a 1.5kb LacZ) in AAVrh.74 capsid, with a genome titer (EGFP targeting) of 1X10^13 VG/ml. This reference is produced using the AAVone system with the pAAVone-AAVrh.74-CMV-EGFP-4.3kb plasmid. AAVrh.74-CMV-EGFP viruses can be simply and efficiently produced by transfection of this single-plasmid into host cells, such us HEK 293 and HEK 293T. AAVrh.74 is a AAV serotype derived from rhesus monkeys. It has gained prominence for its ability to efficiently transduce muscle tissue, making it particularly suitable for gene therapy applications targeting muscle disorders such as Duchenne muscular dystrophy (DMD, ELEVIDYS). AAVrh.74 has been shown to have higher transduction efficiency in skeletal and cardiac muscles compared to other AAV serotypes, such as AAV9, and is being used in clinical trials for its potential to deliver therapeutic genes effectively.

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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.