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

Frank Soldner; Rudolf Jaenisch

doi:10.1016/j.cell.2018.09.010

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

Cell. 2018 Oct 18;175(3):615-632. doi: 10.1016/j.cell.2018.09.010.

Authors

Frank Soldner¹, Rudolf Jaenisch²

Affiliations

¹ The Whitehead Institute for Biomedical Research, 455 Main Street, Cambridge, MA 02142, USA.
² The Whitehead Institute for Biomedical Research, 455 Main Street, Cambridge, MA 02142, USA; Department of Biology, Massachusetts Institute of Technology, 31 Ames Street, Cambridge, MA 02139, USA. Electronic address: [email protected].

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.

Publication types

Research Support, N.I.H., Extramural
Review

MeSH terms

Animals
Central Nervous System Diseases / genetics
Central Nervous System Diseases / therapy*
Gene Editing / methods*
Humans
Stem Cell Transplantation / methods*
Translational Research, Biomedical / methods*

Abstract

Publication types

MeSH terms

Grants and funding