Research

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 (represented above) already contain several powerful properties: binding to a specific region of the genome and performing a precise cut at that site (left, above). This provides the foundation for therapies that may soon be able to correct the genetic defects that give rise to disease. But for the 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 (bottom right, above). Furthermore, we strive to perform targeted delivery of these enzymes, maximizing precision in correcting specific cells, tissues, or organs (top right, above).

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 (represented above) already contain several powerful properties: binding to a specific region of the genome and performing a precise cut at that site (left, above). This provides the foundation for therapies that may soon be able to correct the genetic defects that give rise to disease. But for the 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 (bottom right, above). Furthermore, we strive to perform targeted delivery of these enzymes, maximizing precision in correcting specific cells, tissues, or organs (top right, above).