RNA interference and molecular pathology of selected diseases

: Since many people all around the world are suffering from genetic disorders, modern therapeutic approaches are focused on the search of new pharmaceutical products. These products will be able to act on the gene level, more accurately on the nucleotide sequences themselves. RNA interference (RNAi) is an evolutionary conserved process that is caused by double stranded RNA (dsRNA). MicroRNA (miRNA) and small interfering RNA (siRNA) are the most important dsRNAs, which have been identified so far. Short (19-25 bp) non-coding dsRNAs are responsible for regulation of cellular development, heterochromatin formation and genomic stability in eukaryotes. Most importantly they are able to silence cognate genes. Therefore, they can provide new insights into the gene function and pathway analysis. Furthermore, they are believed to be new potential targets for diagnosis and therapeutics, especially for the treatment of genetic disorders, which can be caused by nucleotides insertions, deletions and translocations.