Nawgen, LLC — Department of Health and Human Services STTR Phase I: 400

Description

Project Summary The CRISPR Cassystem has been developed in recent years for genome editingand it has been rapidly and widely adopted by the scientific communityThe main objective of this grant application is to develop next generation single guide RNAsgRNAwhich is a critical component for CRISPR Casknockout assaysThe sgRNA determines both the efficacy and specificity of CRISPR Casediting activitiesHoweverthe efficacy of sgRNA has not been adequately considered previouslyThe design of potent sgRNAs is highly desiredas inefficient genome editing by CRISPR Caswill inevitably lead to significant waste of resources at the experimental validation stageTo address this challengewe have developed both bioinformatics and experimental methods for improving the design of sgRNAs with high potency and specificityBased on these newly established methodswe propose to further develop a new sgRNA design method to significantly improve the potency of CRIPSR Casknockout assaysOur new genome wide sgRNA assays will lay a solid foundation for further commercial development of CRISPR Casproducts that can be used in a variety of gene knockout applicationssuch as the analysis of individual gene functionhigh throughput functional screen and gene therapy RELEVANCE The CRISPR Cassystem has been developed in recent years for genome editingand it has been rapidly and widely adopted by the scientific communityThe main objective of this grant application is to develop next generation single guide RNAsgRNAwhich is a critical component for CRISPR Casknockout assaysOur new genome wide sgRNA assays will lay a solid foundation for further commercial development of CRISPR Casproducts that can be used in a variety of gene knockout applicationssuch as the analysis of individual gene functionhigh throughput functional screen and gene therapy