Stem cells and regenerative medicine

Abstract There is a great and unmet need for treatments that will deliver restorative solutions to patients with diseases hitherto considered irreparable. Advances in human pluripotent stem cell biology and gene-editing technology offer unprecedented opportunities for both drug discovery and translational therapies that will likely herald a new chapter of regenerative and personalized medicine. Consideration of three major target conditions for regenerative medicine—Parkinson’s disease, heart failure, and diabetes mellitus—emphasizes distinct and common challenges that must be overcome in order to realize the stem cell promise. Novel approaches to induce pluripotency from differentiated somatic cells and targeted genetic manipulation of stem cell populations, along with new insights derived from improved understanding of human pluripotent stem cell biology and increased recognition of endogenous stem cells, offers a range of mechanisms through which stem cells may be therapeutic.