Translating Genome Editing of Stem Cells to the Clinic with Improved Cas9 Specificity


Originally Aired September 20th, 2018

Sponsored by Aldevron

The CRISPR/Cas9 system is a powerful genome-editing tool that can selectively modify and correct an abnormal DNA sequence inside living cells with single-nucleotide precision. It is being used to develop groundbreaking treatments for patients without curative therapies. Nonetheless, the drive to maximize safety of genome editing using this system can call for improved versions of CRISPR/Cas9 and related nucleases that maintain on-target activity, an essential feature for any genome editing approach, while decreasing potentially harmful, off-target nuclease activity.
 
Although there have been several publications describing engineered Cas9 proteins with diminished off-target effects, we found these examples were also significantly lower in on-target activity, probably due to over-engineering, to the extent that their use for clinical application would be limited. We have recently reported a new high-fidelity version of Cas9 that maintains on-target activity and decreases off-target activity by >10-fold in clinically relevant cell types using clinically relevant RNP delivery methods. This new high-fidelity Cas9 variant and how it is being used to develop novel human cell therapeutics will be described in this webinar. In addition to the technical details presented in this webinar, Aldevron will communicate availability and product details of the research grade and GMP high-fidelity version of Cas9 which is marketed under Aldevron’s trade name, SpyFi™ Cas9 Nuclease.

A live Q&A session will follow the presentations, offering you a chance to pose questions to our expert panelists.

Presenter
Matthew Porteus MD, PhD
Associate Professor
Department of Pediatrics and Institute of Stem Cell Biology and Regenerative Medicine
Stanford University
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Presenter
Thomas G. Foti
Vice President
Aldevron, LLC
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Presenter
Moderator: Kevin Davies, PhD
Executive Editor
The CRISPR Journal

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