SafeExo platform for lung-specific drug delivery and gene editing

Using the latest developments in gene editing, direct targeting of oncogenic mutations with CRISPR/Cas can be a highly innovative therapeutic approach. Like all genetic tools, the CRISPR/Cas machinery must be delivered directly to target cells' nuclei to be safe and effective. However, current delivery platforms such as adenovirus, lentiviral vectors, and synthetic delivery methods induce off-target effects and lack tissue tropism toward the lung. Dr. Momen-Heravi's lab has recently developed a novel customizable delivery platform based on engineered exosomes which are devoid of endogenous nucleic acids, called SafeExo-Cas. SafeExo-Cas express endogenous Cas protein and contain targeting moieties toward lung tissue. In this project, she and her group aim to optimize the use of this delivery platform to deliver CRISPR/Cas machinery to lung tissues to treat lung cancer.