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). A first step towards realizing this approach has been published in  Rouet et al. JACS 2018 . 

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). A first step towards realizing this approach has been published in Rouet et al. JACS 2018

 

Publications

Wilson Lab
Receptor-Mediated Delivery of CRISPR-Cas9 Endonuclease for Cell Type Specific Gene Editing
Rouet et al., J Am Chem Soc. 2018 [PDF] [supp. info]
Chemical modification allows a Cas9 RNA-protein (RNP) complex to be selectively taken into cells bearing a liver-associated receptor. This demonstrates the feasibility of using molecular targeting to specify which cells Cas9 will edit, potentially for therapeutic use in vivo

The Promise and Challenge of In Vivo Delivery for Genome Therapeutics
Ross Wilson & Luke Gilbert, ACS Chem Biol. 2018 [PDF]
A review of progress towards genome editing that can cure, prevent, or treat disease, along with a summary of the hurdles that remain. 

All Ross's publications