Viral vices have been center stage throughout the ongoing pandemic, but to the everyday scientist, viruses are essential tools in the research toolbox. Viruses are vectors not of disease, but of gene-edited transcripts, candidate genes, and potential therapeutics. These little vessels help in the grand scientific effort to understand the mechanisms of disease and to mitigate illness.
One such virus, the adeno-associated virus (AAV), is the secret to gene therapy success. AAVs are small viruses of no more than 5,000 kilobases. Scientists often equip AAVs with modified gene sequences and enlist their services to deliver a therapeutic gene to targeted cells. AAV is the vector of choice for gene therapy because of its low immunogenicity, favorable safety profile, and ability to infect numerous cells and tissue types. But manufacturing AAVs is no easy task.
Packaging AAV involves transfecting cells with three different plasmids. One plasmid contains the gene of interest ...
