Journal
JOURNAL OF CONTROLLED RELEASE
Volume 205, Issue -, Pages 120-127Publisher
ELSEVIER SCIENCE BV
DOI: 10.1016/j.jconrel.2014.12.036
Keywords
Engineered nucleases; Genome silencing vs genome editing; Gene, protein and cell therapy; Viral and non-viral delivery systems
Funding
- National Research Foundation of Korea [2014049587]
- Brain Korea 21 plus program [22A20130011095]
- Korean Health Technology R&D project through the Ministry of Health Welfare [HI13C-1938-010014]
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Gene therapy by engineered nucleases is a genetic intervention being investigated for curing the hereditary disorders by targeting selected genes with specific nucleotides for establishment, suppression, abolishment of a function or correction of mutation. Here, we review the fast developing technology of targeted genome engineering using site specific programmable nucleases zinc finger nucleases (ZFNs), transcription activator like nucleases (TALENs) and cluster regulatory interspaced short palindromic repeat/CRISPR associated proteins (CRISPR/Cas) based RNA-guided DNA endonucleases (RGENs) and their different characteristics including pros and cons of genome modifications by these nucleases. We have further discussed different types of delivery methods to induce gene editing, novel development in genetic engineering other than nucleases and future prospects. (C) 2014 Elsevier B.V. All rights reserved.
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