Discovery and analysis of antisense oligonucleotide activity in cell culture

In the past decade antisense oligonucleotides (ASOs) have proven to be a us eful tool for dissection of gene function in molecular cell biology (Koller , E., Gaarde, W. A., and Monia, B. P. (2000) Trends Pharm. Sci., 21, 142-14 8), and validation of gene targets in animal models (Crooke, S. T. (1998) B iotechnol. Gen. Eng. Rev. 15, 121-157), as well as a means for therapeutic treatment of human diseases (Bennett, C. F. (1999) Exp. Opin. Invest. Drugs 8, 237-253). An important step toward usage of ASOs in the described appli cations is identification of an active ASO. This article describes the unde rlying basis and means for achieving this goal in cell culture. (C) 2001 Ac ademic Press.