Accurate determination of AAV vector transduction efficiencies using an image-based direct cell analyzation method
In mammalian cells, DNA is wound around histones and locked away in the nucleus. The most advantageous keys that fit those locks and facilitate nuclear access are found on viruses. The three types of viral vectors most commonly used in the development of gene therapy products are adeno-associated virus (AAVs, 50%), adenovirus (12%), and lentivirus 11%). The effective viral vector transduction and transgene expression in a specific target cell type is a critical early step in the development process.
Multiple AAV serotypes preferentially infect different cell types. The table below (Table 1) shows which serotypes are best in specific tissues . AAV2 is the most studied. It is also possible to mix and match features of different serotypes. For example, taking a capsid from AAV3 and genome from AAV4 would be denoted as AAV3/4. Developing pseudotyped viruses can change vector tropism or which cell type it infects and can also improve transduction efficiency.