High Throughput Cellular Screening for Development of Post-Transcriptonal Gene Silencing Agents against Arbitrary mRNA Targets

The development of RNA based therapies suffers from a bottleneck in identifying lead candidates that  significantly knockdown target RNA in vivo. This is primarily due to the difficulties in predicting the dynamic secondary and tertiary structure of RNA. Currently, researchers begin by using software programs such as MFold and SFold.  However, these programs do not fully take into account the dynamic and complex RNA structure.  Once lead candidates are identified, traditional in vitro screening methods are used to determine RNA knockdown. These methods are costly, time consuming, and contribute to the lack of efficiency found in the preclinical process for RNA based therapeutics.  After years of frustration over the slow pace of traditional methods, researchers at the University at Buffalo have developed a suite of optimizing software and screening tools to address these bottlenecks, with an eye towards increased likelihood of in vivo success. More