University of Colorado Boulder

<p><strong>Overview:</strong></p>
<p>As one of 34 U.S. public research institutions belonging to the prestigious Association of American Universities (AAU)—and the only member in the Rocky Mountain region—University of Colorado Boulder’s (CU Boulder) goal is to directly affect Colorado communities through collaborative research, innovation and entrepreneurship. At CU Boulder, students, faculty and staff are provided with the tools they need to create a community of thinkers and doers who can benefit humanity locally, nationally and abroad.</p>
<p><strong>Research:</strong></p>
<p>Research at CU Boulder encompasses thousands of scholarly, scientific and creative endeavors at any given time, resulting in new knowledge, technologies and creative work that advance the economy, culture and health of Colorado, the nation and the world.</p>
<p>The Research &amp; Innovation Office is focused on creating impact by cultivating and supporting collaboration, transformation and leadership. At the heart of this focus are the diverse contributions of a wide range of world-class experts—working together in new and innovative ways to accelerate ideas through the entire innovation lifecycle, from idea to impact.</p>
<p><strong>Industry Partnerships:</strong></p>
<p>Ranked as one of the nation’s top 20 entrepreneurial universities and the #2 most innovative tech hub in the U.S., CU Boulder has granted 140 license and option agreements to businesses in the past five years from CU Boulder discoveries.</p>
<p>With expertise in patents, copyrights and licensing, the CU Boulder Tech Transfer Office helps translate discovery into impact through transparent, flexible, best practice intellectual property management services, and connect campus researchers with a variety of commercialization programs in the university and the community. </p>


<h1 id="background">Background</h1>
<p>One version of the CRISPR/Cas system, CRISPR/Cas12a, has been modified to provide useful tools for editing genomes. Cas12a-type nucleases have gained popularity due to their versatility and simplicity as compared to Cas9. These systems are useful but have some important limitations regarding efficiency and accuracy of targeted editing, resulting in impediments when used for commercially relevant applications. Therefore, a need exists for improved nucleic acid guided nucleases for directed and accurate editing with improved efficiency.</p>
<h1 id="technology-overview">Technology Overview</h1>
<p>Researchers from the University of Colorado have developed a method of engineering chimeric Cas12a-type nucleases for improved targeted gene editing. This method was used to create 560 synthetic Cas12a-type nucleases, resulting in nucleases with diverse PAM preferences and on/off-targeting specificity. In addition to advantages afforded by Cas12a (AT rich PAM, staggered cuts, and simpler multiplex editing), these chimeric nucleases have capabilities to fit specific gene editing needs.</p>
<p>Further Details</p>
<p>Liu, R.M., Liang, L.L., Freed, E. et al. Synthetic chimeric nucleases function for efficient genome editing. Nat Commun 10, 5524 (2019). <a href="https://pubmed.ncbi.nlm.nih.gov/31797930/">https://doi.org/10.1038/s41467-019-13500-y</a></p>
<h3 id="-stage-of-development-"><strong>Stage of Development</strong></h3>
<ul>
<li>Technology Readiness Level (TRL): 4.</li>
</ul>
<h1 id="benefits">Benefits</h1>
<ul>
<li>Alternative PAM preferences</li>
<li>Higher specificity to wild-type sequences</li>
<li>Activity demonstrated in yeast and HEK293T cells</li>
<li>Multiple nuclease options to suit specific genome editing needs</li>
<li>Methodology can be used to create further tailored nuclease</li>
</ul>
