Antisense Approaches in Analyzing the Functional Role of Proteins in the Central Nervous System

Along with the emergence and development of the new field of “proteomics,” neuroscientists together with other life scientists have been offered the greater opportunity to clone and sequence new biologically active proteins and to assess alterations in protein expression at both mRNA and protein levels in response to a variety of experimental manipulations (1 ). As proteins serve as important elements and molecules in cellular structures, receptors, transporters, factors, enzymes, and so on, functional roles of these various proteins largely determine cellular physiology. To assess the role of proteins, especially receptors and enzymes, pharmacological antagonists/inhibitors have been used as effective tools for decades. However, although these pharmacological tools possess potent antagonistic actions, the selectivity against their specific targets is relative and sometimes far from satisfactory. Moreover, the antagonistic properties of these tools, which are usually characterized in vitro, may not be reflected in vivo. Lack of selective agents at all is another dilemma for analyzing the function of some proteins, such as structural proteins, transcription factors, and so forth.