AIDS is a fatal disorder of the immune system caused by the HIV and is a widespread epidemic. The long term objective of this work is to treat human AIDS patients with a novel oligonucleotide (T30177) that has been shown to have anti-HIV activity. We will perform preclinical animal pharmacology studies, including pharmacokinetic/tissue disposition and toxicity studies in mice. One of these aims includes investigating a novel in vivo route of administration of oligonucleotides. Although there are a number of drugs that are currently available for the treatment of AIDS, these drugs have limited effectiveness and only delay the progression to death of AIDS patients. There is a great need for the development of more effective and novel drugs for the treatment of AIDS. T30177 may provide significant advantages over presently available HIV drugs because it has a novel mechanism of action, it is active against AZT-resistant HIV, and it has been shown in pilot animal studies to have a long half-life, good stability, and low toxicity.National Institute of Allergy and Infectious Diseases (NIAID)
