AAV-PHP.eB Reference-4.3kb

The AAV-PHP.eB reference-4.3kb material contains a 4.3kb AAV-CMV-SV40I-EGFP-WPRE-hGHpolyA genome (with a 1.5kb LacZ) in AAV-PHP.eB capsid, with a genome titer (EGFP targeting) of 1X10^13 VG/ml. This reference is produced using the AAVone system with the pAAVone-AAV-PHP.eB-CMV-EGFP-4.3kb plasmid. AAV-PHP.eB is an engineered variant of the AAV9 serotype that was developed to improve the transduction efficiency of the central nervous system (CNS), particularly in mice. It was designed through directed evolution to cross the blood-brain barrier (BBB) with significantly enhanced efficiency compared to the natural AAV9 serotype, making it a valuable tool for gene delivery to the brain and spinal cord.

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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.