title: A cut above the rest: targeted genome editing technologies in human pluripotent stem cells.
abstract: Human pluripotent stem cells (hPSCs) offer unprecedented opportunities to study cellular differentiation and model human diseases. The ability to precisely modify any genomic sequence holds the key to realizing the full potential of hPSCs. Thanks to the rapid development of novel genome editing technologies driven by the enormous interest in the hPSC field, genome editing in hPSCs has evolved from being a daunting task a few years ago to a routine procedure in most laboratories. Here, we provide an overview of the mainstream genome editing tools, including zinc finger nucleases, transcription activator-like effector nucleases, clustered regularly interspaced short palindromic repeat/CAS9 RNA-guided nucleases, and helper-dependent adenoviral vectors. We discuss the features and limitations of these technologies, as well as how these factors influence the utility of these tools in basic research and therapies.
authors.affiliation: From the Gene Expression Laboratory, Salk Institute for Biological Studies, La Jolla, California 92037 and.
authors.forename: Mo
authors.initials: M
authors.lastname: Li
authors.forename: Keiichiro
authors.initials: K
authors.lastname: Suzuki
authors.forename: Na Young
authors.initials: NY
authors.lastname: Kim
authors.forename: Guang-Hui
authors.initials: GH
authors.lastname: Liu
authors.forename: Juan Carlos
authors.initials: JC
authors.lastname: Izpisua Belmonte
mesh_terms: D003851:Deoxyribonucleases / Q000378:metabolism; D005818:Genetic Engineering / Q000379:methods*; D015894:Genome, Human / Q000235:genetics*; D006801:Humans; D039904:Pluripotent Stem Cells / Q000378:metabolism*; D017393:RNA Editing / Q000235:genetics*
chemical_list: D003851:Deoxyribonucleases
keywords: CRISPR/CAS9; Embryonic Stem Cell; Gene Therapy; Genome Editing; HDAdV; Induced Pluripotent Stem Cell (iPSC); Stem Cells; TALEN; ZFN