ABSTRACT
The FDA approved the first antisense therapeutic, fomivirsen, a treatment for cytomegalovirus retinitis in AIDS patients. An antisense compound targeted to the gag region of human immunodeficiency virus was pulled from clinical trials due to the resulting decreases in platelet counts in 30% of the patients after 10 days of daily administration. Antisense oligonucleotides have the potential to selectively inhibit the expression of any gene with a known sequence. Targeted delivery of antisense oligonucleotides is needed to minimize side effects and maximize oligonucleotide concentration in target cells. The potential applications of antisense oligonucleotides to the treatment of disease are vast. The activity of the oligonucleotide therapeutic is potentiated by traditional drugs such as ganciclovir. Additional studies suggested, however, that the oligonucleotide acts not only via an antisense mechanism, though complementarity to the target was required for maximal inhibitory capacity.
