AAV9 Reference-4.3kb

The AAV9 reference-4.3kb material contains a 4.3kb AAV-CMV-SV40I-EGFP-WPRE-hGHpolyA genome (with a 1.5kb LacZ) in AAV9 capsid, with a genome titer (EGFP targeting) of 1X10^13 VG/ml. This reference is produced using the AAVone system with the pAAVone-AAV9-CMV-EGFP-4.3kb plasmid. AAV9 is one of the most widely used adeno-associated virus serotypes, especially known for its ability to efficiently transduce a broad range of tissues, including the central nervous system (CNS), liver, heart, and muscle. AAV9 has garnered considerable attention in the field of gene therapy due to its unique ability to cross the blood-brain barrier (BBB) and efficiently transduce neurons following systemic administration. This distinctive feature makes it particularly well-suited for both peripheral and central nervous system (CNS) gene therapy applications. A prominent example of its use is in Zolgensma (Onasemnogene Abeparvovec), a groundbreaking treatment for spinal muscular atrophy (SMA). SMA is a severe genetic disorder characterized by the loss of motor neurons and progressive muscle weakness.

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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.