Unlocking Biology's Potential

During the 11 years since its completion, the human genome project paved the way for many advances in science and medicine, ranging from deeper understanding of disease development and progression, to expanding bioinformatic and diagnostic tools, to development of novel therapeutics for cancer, metabolic disorders, and other ailments. A direct application of the vastly expanded genetic information is the development of small interfering RNAs (siRNAs) and microRNAs as targeted therapeutics for cancers, skin diseases, and other disorders of human health. These readily synthesized and specific modulators of messenger RNA expression have a huge potential for selective and specific treatment. However, the complications of delivering these biologicals to the cytoplasm of target cells stands in the way of harvesting these therapeutic fruits of the human genome project. This session will examine the problems encountered by academia and the pharmaceutical industry and discuss cutting-edge and current efforts to circumvent these barriers. The speakers, Daniel D. von Hoff (Translational Genomics Research Institute and U.S. Oncology), Anil K. Sood (University of Texas MD Anderson Cancer Center), and Patrick S. Stayton (University of Washington), are experts in all areas associated with these problems and will provide a broad perspective on the development of genome project–induced new medicines, discuss current efforts to deliver siRNAs in animals and humans, and share advances in polymeric materials and nanotechnology that enable the delivery of siRNA in patients.