Regenerative Medicine 2006

T he Greek Titan, Prometheus, is a fitting symbol for regenerative medicine. As punishment for giving fire to Humankind, Zeus ordered Prometheus chained to a rock and sent an eagle to eat his liver each day. However, Prometheus' liver was able to regenerate itself daily, enabling him to survive. The scientific researchers and medical doctors of today hope to make the legendary concept of regeneration into reality by developing therapies to restore lost, damaged, or aging cells and tissues in the human body. This report features chapters written by experts in several areas of enormous potential for regenerative medicine. explain the basic features of embryonic stem cells, how they are being used in research, and how they may lead to human therapies. Drs. Jos Domen, Amy Wagers, and Irving Weissman describe the historical origins of blood-forming stem cell research, basic features of these adult stem cells, progress on using these cells for human therapies, and future possibilities. Dr. David Panchision explores ways to use cell-based therapies to restore lost function in the human nervous system. Dr. Thomas Zwaka explains how stem cells may be used for gene therapy, and Dr. Mark L. Rohrbaugh explains the current state of intellectual property issues associated with research using human embryonic stem cells. i INTRODUCTION [This page intentionally left blank] ii H uman embryonic stem (ES) cells capture the imagination because they are immortal and have an almost unlimited developmental potential (Fig. 1.1: How hESCs are derived). After many months of growth in culture dishes, these remarkable cells maintain the ability to form cells ranging from muscle to nerve to blood — potentially any cell type that makes up the body. The proliferative and developmental potential of human ES cells promises an essentially unlimited supply of specific cell types for basic research and for transplantation therapies for diseases ranging from heart disease to Parkinson's disease to leukemia. Here we discuss the origin and properties of human ES cells, their implications for basic research and human medicine, and recent research progress since August 2001, when President George W. Bush allowed federal funding of this research for the first time. Embryonic stem cells are derived from embryos at a developmental stage before the time that implantation would normally occur in the uterus. Fertilization normally occurs in the oviduct, and during the next few days, a series of cleavage divisions occur as the embryo travels down the …