AAV Reference Standards
A safe strategy for gene therapy.
Adeno-associated virus (AAV) is a virus that can be engineered to transport DNA of interest to target cells, without any viral genes. There has been a revival in gene therapy efforts, particularly in research and clinical applications, at least partially powered by the progress in understanding of AAV. The potential of recombinant AAV particles is sky-high and is proven to be one of the current safest strategies for gene therapy.
Arguably the greatest challenge in AAV production is that AAV packages a significant number of viral particles without gene payloads. Even after purification procedures, there will still be empty capsids. Therefore, a well-characterized AAV standard with an accurate full/empty ratio is needed to distinguish the final vector production and accurately compare AAV success.
High quality and high purity empty capsid standards are used as reference materials in an extensive range of applications for example, qPCR, ELISA, HPLC etc.
AMSBIO provide extensively analyzed AAV reference materials with viral particle concentration and full/empty ratio. These are provided in the form of 5 serotypes: AAV1, AAV2, AAV5, AAV6, AAV9
Both full and empty capsids are available for many serotypes.
Our reference materials are well-characterized, including bioburden, endotoxin, mycoplasma testing, silver staining (Figure 1), ELISA, qPCR and transmission electron microscopy (TEM) (Figure 2) and are quantified based on the reference standard material from ATCC.
The stability of our reference material will be tested in an ongoing manner to ensure reliability.