Nawgen, LLC — Department of Health and Human Services STTR Phase I: 172

Description

DESCRIPTION provided by applicant RNA interference RNAi is a small RNA guided gene silencing process within living cells The RNAi technique is widely used in both biological research and clinical applications because it has the ability to knockdown essentially any gene of interest However one major unresolved issue in RNAi studies is non specific gene silencing It is well known that in addition to the single intended gene target many unintended targets are also simultaneously silenced Thus there is an urgent need to develop new methods for improving RNAi specificity RNAi specificity and potency are determined by the small interfering RNA siRNA in the silencing complex We propose the hypothesis that gene silencing specificity can be significantly improved with rational siRNA design We have previously developed a machine learning algorithm for the design of widely distributed commercial siRNAs Based on this commercial success we propose to further develop a new method for genome wide design of next generation siRNAs with significantly reduced off target effects The new design method will lay a solid foundation for further commercial development of siRNA products that can be used in a variety of RNAi based applications PUBLIC HEALTH RELEVANCE RNA interference RNAi is a small RNA guided gene silencing process within living cells RNAi has many diverse applications as it can suppress almost any human genes by recognizing the gene sequence We propose to remove a major hurdle in RNAi studies by genome wide design of siRNAs with significantly improved gene silencing specificity