Projects
CNS drug target validation using RNA interference
CNS drug target validation using RNA interference Disorders of the central nervous system (CNS) are complex, poorly understood, and debilitating diseases. The unmet medical need for new CNS drugs is extremely high. Genetic and functional genomics studies have identified novel drug targets with considerable therapeutic potential that will be validated in this project. One of the major hurdles facing the pharmaceutical industry is the timeconsuming process of CNS drug target validation in vivo, which is needed to confirm clinical relevance. RNA-based technology (e.g. RNAi and exon skipping) is an alternative for the selective and rapid validation of novel targets in animals and it can also be used in a clinical setting. This project will shorten the timeconsuming process of target validation by using RNA-based technology in vivo. In 2008, tools (viral vectors and genetically modified rodents) to validate the first targets were generated and novel targets to downregulate using RNAi were identified. The effect of knockdown of these targets is under investigation.
Partners: Abbott, Academic Medical Center (AMC) Amsterdam, Leiden University, Prosensa, the University Medical Center Utrecht
Full project title: Rapid in vivo CNS drug target validation and therapeutic potential using RNA interference