Stem Cells, Genome Editing, and the Path to Translational Medicine

Abstract

The derivation of human embryonic stem cells (hESCs) and the stunning discovery that somatic cells can be reprogrammed into human induced pluripotent stem cells (hiPSCs) holds the promise to revolutionize biomedical research and regenerative medicine. In this Review, we focus on disorders of the central nervous system and explore how advances in human pluripotent stem cells (hPSCs) coincide with evolutions in genome engineering and genomic technologies to provide realistic opportunities to tackle some of the most devastating complex disorders. Advances in stem cell biology are paving new paths toward their use in the clinic, especially toward understanding and treating neurological and neurodegenerative disease.