Antisense oligonucleotides: local delivery enhances their therapeutic potential

Abstract

The field of antisense oligonucleotides (ASONs) has now matured to the point that much of the original optimism regarding their therapeutic potential has been rekindled. The first antisense to reach the commercial stage, fomivirsen (Vitravene™) for cytomegalovirus (CMV) retinitis in immunocompromised patents, may be prescient for future successful members of this new therapeutic class in that it clearly illustrates the utility of ASONs when the problem of delivery is solved. The simplest way to solve the delivery problem is to administer ASONs directly, i.e., locally, to the target tissue. Fomivirsen is approved for direct intravitreal injection. This brief review examines recent work on local delivery of ASONs to treat a variety of human diseases.

• antisense oligonucleotides
• cervical
• ex vivo
• intravitreal
• local delivery
• respirable
• topical