Viral vectors have been used for in vivo delivery of CRISPR/Cas9. However, it’s applications have been limited by the native tropism of the viral vector. To better utilize in vivo editing, methods must be developed to accomplish efficient, selective and coordinated delivery of CRISPR/Cas9 components to the relevant target cell.
Dr. David Curiel at Washington University School of Medicine has developed a method to perform in vivo editing in specific tissue types by combining targeted adenovirus with CRISPR/Cas9. This is made possible due to the unique capacities of adenovirus. The packaging capacity of adenovirus allows vector incorporation of all CRISPR/Cas9 elements ensuring efficient coordinated functionality. Adenovirus’s ability to allow tropism modification makes in vivo targeting feasible. Thus, targeted adenovirus embodies the unique set of attributes required for the key endpoint of targeted in vivo gene editing.
- Targeted in vivo gene editing
- Safe Harbor locus