Abstract

siPOOLs – Application in High Content Screening and Target Validation

Catherine Goh, Head of Product Development and Marketing, siTOOLs Biotech GmbH

RNA interference (RNAi) has numerous advantages as a gene disruption tool. It is easily and broadly applied, reversible and dose-dependent, producing results rapidly with limited expenditure on resources. These drug-like properties have made it the tool of choice for numerous functional genomic screens.  However, a major drawback of RNAi is the non-specificity of silencing RNAs (siRNA) which generates numerous off-target effects. These off-target effects dominate most published RNAi screens and limit their yield to a small number of strong responders or already well-established genes.   siPOOLs are high complexity pools of 30 carefully selected siRNAs. The very low concentration of each siRNA administered greatly reduces off-target effects while the cooperative activity of multiple siRNAs increases robustness and reproducibility of target gene knock-down.  With safety, speed and efficiency being a priority in drug development, siPOOLs enable rapid functional genomic screening while reducing the risks of capturing false leads. Proof-of-concept data and applications towards high content screening and target validation will be shared and discussed.