Therapeutic potential of small interfering RNA for central nervous system diseases

RNA interference (RNAi) is an evolutionarily conserved mechanism to degrade messenger RNA (mRNA) that has sequence homology to small double-stranded RNA (dsRNA). It is believed that the normal biological function of RNAi is to protect the host cells from RNA viruses and transposons, which can jeopardize the genome. Production of dsRNA in the host cell signals a series of events that ultimately results in the degradation of complementary mRNA. Recently, small interfering RNA (siRNA) has been used to study the function and significance of a vast number of genes in a variety of cell types. In the future, siRNA may have tremendous potential as gene-specific therapeutic agents for the treatment of many diseases. We discuss the potential role of siRNA as a novel therapeutic strategy for several central nervous system (CNS) diseases.