Therapeutic genome editing: prospects and challenges

Available in PMC 2015 July 06Recent advances in the development of genome editing technologies based on programmable nucleases have substantially improved our ability to make precise changes in the genomes of eukaryotic cells. Genome editing is already broadening our ability to elucidate the contribution of genetics to disease by facilitating the creation of more accurate cellular and animal models of pathological processes. A particularly tantalizing application of programmable nucleases is the potential to directly correct genetic mutations in affected tissues and cells to treat diseases that are refractory to traditional therapies. Here we discuss current progress toward developing programmable nuclease–based therapies as well as future prospects and challenges.Robert MetcalfeSimons FoundationMerkin Family Foundation for Stem Cell ResearchNational Science Foundation (U.S.) (NSF Waterman Award)National Science Foundation (U.S.) (NSF Graduate Research Fellowship, grant number 1122374)National Institute of General Medical Sciences (U.S.) (Award number T32GM007753)National Institutes of Health (U.S.) ((NIH) Director’s Pioneer Award (DP1-MH100706))W. M. Keck FoundationNational Institute of Neurological Disorders and Stroke (U.S.) (NIH Transformative R01 grant (R01-NS 07312401))Damon Runyon Cancer Research FoundationSearle Scholars ProgramEsther A. & Joseph Klingenstein Fund, Inc.Vallee Foundatio