ABSTRACT

Initial efforts in the creation of antisense therapeutics have focused on fundamental questions such as safety, efficacy, and technology costs, as would be expected for any emerging technology with a potential to create a new therapeutic paradigm. Clinical proof of efficacy has been established with the approval of the first antisense oligonucleotide therapeutic, Vitravene™ (fomivirsen), indicated for the local treatment of cytomegalovirus (CMV) retinitis in patients with AIDs [1].