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Antisense oligonucleotide technology: from EST to therapeutics
1Department of Haematology, Liverpool University, Life Science Building, Crown Street, Liverpool L69 7ZB, UK. giles@liv.ac.uk
Abstract:
The ability to selectively inhibit gene expression with antisense oligonucleotides could have profound effects upon future biological research, including functional genomics, and the ways in which many diseases are treated. This review describes cell culture-based research pertaining to the selection of optimal antisense effector sequences, their desired and non-antisense mechanisms of action and their delivery into the appropriate intracellular compartments. Preclinical and clinical use of antisense technology is examined and probable future directions that this may take are discussed. Overall, the current state of antisense oligonucleotide research described here promises a highly productive future for this technology.