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 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  (see summary below).

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 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 (see summary below).

 

Publications

Wilson Lab

Clinical Applications of CRISPR-based Genome Editing and Diagnostics
Dana Foss, Megan Hochstrasser, Ross Wilson; Transfusion 2019 [PDF]
We review CRISPR’s transition into the clinic, via ex vivo therapies and emerging diagnostic tools.

Engineering CRISPR-Cas9 RNA–Protein Complexes for Improved Function and Delivery
Romain Rouet, Lorena de Oñate, Jie Li, Niren Murthy, Ross Wilson, CRISPR J. 2018 [PDF]
We summarize recent progress in the engineering of Cas9 RNA-protein (RNP) complexes for cellular delivery, improved function, and more.

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. 

Emerging Strategies for Genome Editing in the Brain
Dana Foss & Ross Wilson; Trends Mol Med. 2018 [PDF]
This Spotlight article describes nanoparticle-driven delivery of Cas9 RNP enzymes and compares/contrasts this approach to virally mediated delivery. 

All of Ross's publications