Antisense technology toxicity

The potential of the chemically modified nucleic acid molecules has been proven by in vitro studies however, the in vivo therapeutic applicability of these molecules seems to be unsatisfactory because of their possible toxic effects (largely unknown) and adverse bioavailability. In this view, both antisense and transfection technologies require reliable and efficient systems for their delivery into target cells. On the basis of this consideration, the development of an efficient nucleic acid delivery system represents one of the key steps for these therapeutic agents, which are necessary for a practical clinical utilization of natural or unnatural oligonucleotides. 

Levin AA, Henry SP, Monteith D, Templin M. Toxicity of antisense oligonucleotides. In Crooke ST, ed. Antisense Drug Technology. New York Dekker, 2001 201-67. 

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