The concept and application of antisense oligonucleotides

Since the identification of the double-stranded DNA helix by Watson and Cri ck in 1953, the knowledge of nucleotide structure and function has been an important potential tool in the study and therapy of disease. There is rece nt clinical evidence that antisense oligonucleotides may be important thera peutic compounds in the clinical therapy of a range of diseases, including infection (viruses and bacteria), oncology, and inflammation. Our laborator y-based understanding of antisense oligonucleotide activity has provided a foundation for their use in several human diseases. Potentially relevant ap plications include inflammatory bowel disease therapy, psoriasis, transplan tation, rheumatoid arthritis, cytomegalovirus retinitis, hepatitis C, and s olid tumor therapy. Here we will outline these applications as well as our ongoing clinical trials for Crohn's disease.