Year: 2013
Type: review
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.
Source: Journal of Biological Chemistry
Language: English
Subfield: Molecular Biology
Domain: Life Sciences
Open Access status: hybrid
Estimated APC paid: $3,430
Funders: Ellison Medical Foundation, Glenn Foundation for Medical Research, Leona M. and Harry B. Helmsley Charitable Trust, California Institute of Regenerative Medicine, Chinese Academy of Sciences, Fondation pour la Recherche Médicale, Thousand Young Talents Program of China, National Heart, Lung, and Blood Institute, National Laboratory of Biomacromolecules
OpenAlex ID: W2112959936