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Gene editing with RNA-guided nucleases based on the CRISPR bacterial defence system (clustered regularly interspaced palindromic repeats) has revolutionized both basic science and therapeutic applications. However, CRISPR associated (Cas) nucleases are not optimized for all types of gene-editing applications. In particular, the double-strand breaks made in DNA by the Cas proteins are imperfectly repaired by cellular DNA repair pathways that lead to a spectrum of gene-editing outcomes. The goal of this project is to develop new dual-cleaving RNA guided nucleases that generate defined gene-editing outcomes and that can target sequences not accessible by commonly used Cas9 nucleases. Given the wide-spread adoption of gene-editing technologies, the new dual-cleaving nucleases would provide alternative and improved gene-editing tools for basic scientific studies by academic and industry researchers. For Specific Biologics, this partnership supports future downstream therapeutic use of new dual-cleaving nucleases to target genetic mutations that cannot be targeted with current gene editors.
David Edgell
Specific Biologics Inc
Life Sciences
Professional, scientific and technical services
The University of Western Ontario
Accelerate
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