RNAi has become a powerful tool for rapidly evaluating the biological impact of suppressing the expression of an individual gene. Specific applications include the following:
Mechanism of action studies
Universal gene expression analysis tools, such as reverse-transcription qPCR (RT-qPCR) arrays, and hybridization-based microarrays, enable the rapid identification of gene expression events that are linked to disease states or treatment regimens. These types of studies are categorized as reverse genetics approaches. RNAi has become a tool of choice for validating the functional relevance of the gene products identified in such reverse genetics screens. QIAGEN has established the FlexiTube and FlexiPlate siRNA product lines to serve the needs of such applications.
Drug target validation studies
Once a putative drug target has been identified via chemical, biochemical, molecular biological, or cell-based screening, RNAi technology has become a widely-used tool for subsequent drug target validation studies.
Perform high throughput, genome-wide functional screening
siRNA libraries, in combination with robust cell-based assays, are utilized as a screening tool, to interrogate the transcriptome, in search of gene expression events that are relevant to an important phenotypic change. FlexiPlate siRNA offers a suitable tool to perform targeted RNAi screens focusing e.g. on specific pathways or specific scientific questions..
Preclinical and early phase clinical studies are ongoing to evaluate the efficacy of siRNA-based anti-viral therapies, as well as treatments for metabolic diseases and age-related macular degeneration (AMD).