Advantages of antisense drugs for the treatment of oral diseases.

Abstract

For almost two decades, antisense oligonucleotides (AS-ON) have been used successfully to suppress and regulate gene expression in vitro and in vivo. They are, meanwhile, well established to serve as molecular tools for several biologic applications, from the study of single gene functions up to complex target gene validations. Based on an at least theoretically simple mode of action, the sequence-specific inhibition of mRNA functions after complex formation by Watson-Crick base pairing and presumably enzymatic degradation of the target mRNA, they obviously carry a high therapeutic potential for the treatment of human diseases. In recent years, a remarkable number of clinical trials have been initiated and performed to evaluate the therapeutic usefulness of antisense technology. However, after the successful development of the first antisense-based drug Vitravene (Isis Pharmaceutical Inc., Carlsbad, CA) in 1998, no second product has appeared on the market to date. Here, we describe substantial advantages for the development of antisense-based drugs against less severe oral diseases that represent novel but highly promising application fields of the technology.