Therapeutic Potential of Small Interfering RNA for Brain Diseases

摘要: RNA interference (RNAi) is an evolutionarily conserved mechanism found in all eukaryotes to degrade messenger (mRNA) that has sequence homology short double-stranded (dsRNA). It believed the usual biological function of RNAi safeguard host cells from viruses and transposons, which can endanger genome. Expression dsRNA cell signals a cascade intracellular events generate small interfering (siRNA) segments mediate degradation complementary mRNA subsequent failure express protein. Recently, engineered siRNA been used study normal significance vast number genes variety types, as well role specific disease pathogenesis. Thus, therapeutic potential specifically suppress expression proteins associated with processes. The exquisite specificity siRNA-mediated gene suppression provides opportunity target single point mutations hereditary disorders, unique pathogenic tumor malignancies, are critical for mediating pathology many diseases. fact functions at transcription level means virtually any be targeted suppression, providing strategy develop therapeutics neurological diseases, have no effective therapies.