The use of short, synthetic nucleic acids as potential therapeutics was proposed more than 3 decades ago. However, improvements in the design and synthesis of nucleic acids have recently piqued the interest of researchers and physicians searching for alternative approaches for treating diseases that do not conform to traditional drug discovery programs. The advent of novel modified nucleic acids with better efficacy, stability, and cell penetrance combined with advancements in various delivery systems and methods, including intrathecal administration to allow direct entry to the central nervous system, provide sufficient rationale to cultivate the therapeutic capabilities of synthetic nucleic acids for neurological diseases.
Butler JS, Napierala M. New Reasons to Pursue the Therapeutic Potential of Synthetic Nucleic Acids for Neurological Diseases. JAMA Neurol. 2016;73(10):1175-1177. doi:10.1001/jamaneurol.2016.2571