Format

Send to

Choose Destination
See comment in PubMed Commons below
J Biol Chem. 2014 Feb 21;289(8):4594-9. doi: 10.1074/jbc.R113.488247. Epub 2013 Dec 20.

A cut above the rest: targeted genome editing technologies in human pluripotent stem cells.

Author information

1
From the Gene Expression Laboratory, Salk Institute for Biological Studies, La Jolla, California 92037 and.

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.

KEYWORDS:

CRISPR/CAS9; Embryonic Stem Cell; Gene Therapy; Genome Editing; HDAdV; Induced Pluripotent Stem Cell (iPSC); Stem Cells; TALEN; ZFN

PMID:
24362028
PMCID:
PMC3931021
DOI:
10.1074/jbc.R113.488247
[Indexed for MEDLINE]
Free PMC Article
PubMed Commons home

PubMed Commons

0 comments
How to join PubMed Commons

    Supplemental Content

    Full text links

    Icon for HighWire Icon for PubMed Central
    Loading ...
    Support Center