Dr Niels Montano Frandsen, Exiqon

miRNAs have been the subject of intense investigation since
their discovery a decade ago. This is due to their central role in regulation in gene expression and their implication in a growing number of important diseases such as cancer, heart failure, diabetes and immune related diseases. Recent discoveries that miRNAs are present and highly stable in serum/plasma and other biofluids has opened up yet another avenue of research. Little is known about the role of circulating miRNAs, but there is speculation that they may represent a novel endocrine system important for transmission of signals in the body. In addition miRNAs may represent a goldmine as noninvasive biomarkers of diseases and drug treatment.

Due to their small size the study of microRNAs is especially challenging. Exiqon has developed several Locked Nucleic Acid (LNA?) based technological platforms to detect microRNAs and uncover their functions. We have developed a highly sensitive LNA? -based quantitative PCR system that opens new possibilities for genome profiling of miRNAs in limited clinical samples with very low RNA content such as blood serum/plasma. We will present results from our diagnostic development programme that suggests that early detection of colorectal cancer based on miRNA profiling of plasma may be feasible.

The second part of the presentation will focus on the use of LNA oligonucleotides as highly versatile tools for functional analysis of proteins, mRNA processing, miRNAs and other ncRNAs. These tools are routinely used in cell cultures, but are now increasingly also being successfully used in live animals.