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a Pommeranian Medical Academy, 71245 Szczecin, Poland b Division of Hematology/Oncology, Department of Medicine and Abramson Family Cancer Research Institute, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania 19104, USA c Department of Bioorganic Chemistry, Uppsala University, Biomedical Center, S-751 23 Uppsala, Sweden d Department of Chemistry, McGill University, Montreal, QC, Canada H3A 2K6
Key Words: leukemia • lymphoma • antisense • oligonucleotides
Address for correspondence: Alan M. Gewirtz, M.D., Division of Hematology/Oncology, Department of Medicine and Abramson Family Cancer Research Institute, University of Pennsylvania School of Medicine, Philadelphia, PA 19104. Voice: 215-898-4499; fax: 215-573-2078. e-mail: gewirtz{at}mail.med.upenn.edu
Our work is motivated by the belief that RNA targeted gene silencing agents can be developed into effective drugs for treating hematologic malignancies. In many experimental systems, antisense nucleic acids of various composition, including antisense oligodeoxynucleotides (AS ODNs) and short interfering RNA (siRNA), have been shown to perturb gene expression in a sequence specific manner. Nevertheless, our clinical experience, and those of others, have led us to conclude that the antisense nucleic acids (ASNAs) we, and others, employ need to be optimized with regard to intracellular delivery, targeting, chemical composition, and efficiency of mRNA destruction. We have hypothesized that addressing these critical issues will lead to the development of practical and effective nucleic acid drugs. An overview of our recent work which seeks to addresses these core issues is contained within this review.
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