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RESEARCH
The Wilson lab focuses on engineering genome editing enzymes for cell-targeted delivery. CRISPR-based genome editing technology has rapidly transformed biomedical research and shows great promise for the development of novel therapeutic applications. Enzymes such as Cas9 already contain several powerful properties: binding to a specific region of the genome and performing a precise cut at that site. This provides the foundation for therapies that may soon be able to correct the genetic defects that give rise to disease. But for this promise to be fully realized, therapeutic enzymes must be delivered to cells safely and efficiently. Our laboratory is working to develop genome-editing enzymes that are readily internalized by cells. Furthermore, we strive to perform targeted delivery of these enzymes, maximizing precision in correcting specific cells, tissues, or organs. A first step towards realizing this approach has been published in Rouet et al. JACS 2018.

Recent advances in genetic and cell-based technology have demonstrated the transformative power of next-generation therapeutics. However, these treatments are incredibly expensive, and are often only available at a handful of cutting-edge research hospitals. Tragically, there are millions of people in the developing world who would benefit from such treatments, but deployment is currently impractical. The Wilson Lab’s efforts are motivated by the pressing need to improve access to life-changing technologies. A central goal of our work is to find affordable and straightforward new ways to genetic medicines, enabling cures globally.

The Gene Editing / Gene Therapy Seminar Series features leading academic, translational, and investor speakers in e-talks through the summer and fall.