Targetable vectors have shown great promise in the field of gene therapy, allowing accurate and effective delivery of therapeutic transgenes to any pre-selected tissue or cell type in vivo, without affecting neighboring non-target cells. A variety of strategies have been developed for different viruses (and their derivative vectors) to introduce their nucleic acid payload into target cells. Regardless of the targeting strategy used, there is a need to evaluate their targeting capabilities after initial proof studies and vector construction are complete, providing valuable information for optimizing gene therapy.
In order to evaluate new targeting strategies in preclinical drug development for rare diseases, we have developed several models that reflect the human condition to varying degrees including primary cells, tissue explants systems, and transgenic animals. In addition to this, the use of imaging technologies for vector targeting assessment appears particularly promising, offering the ability to monitor vectors in vivo in real time without sacrificing animal models.