Ben Berkhout, University of Amsterdam, Netherlands
Fatah Kashanchi, George Washington University, USA
The simplicity and broad applicability of targeted and programmable genome editing approaches, including but not limited to those based on CRISPR-Cas9, raise the possibility of a fundamentally new way to treat a variety of infections, as well as numerous therapeutic strategies for common diseases.
However, many challenges need overcoming before such techniques could be widely used in the clinic. Current issues of bottleneck in the field are complex; however, few stand out as particularly challenging. They include lack of optimized genome editors, specifically targeted delivery systems, reduced immunogenicity, improved or expanded cell-type targeting, simplification or increased scalability of production, transient or regulatable expression of genome editors, directed evolution, and selection methodologies to guide the identification of complexes with novel enzymatic activities, approaches to increase the frequency of HDR within target regions, while reducing non-specific events and substrate specificities and more predictive animal models and studies.
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Deadline for submissions: 31 July 2022