Abstract

One of the most exciting aspects of antisense therapeutic agents is their remarkable pharmacologic specificity. Because antisense activity depends on Watson and Crick base-pairing rules, sequence specificity ensures that antisense agents have high selectivity for the intended mRNA target. The selective inhibition of the expression of disease-related genes is the pharmacologic equivalent of laser surgery, well aimed and presumably with minimal inadvertent effects. This specificity suggests that these agents will have favorable therapeutic indices.

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