| Literature DB >> 25553825 |
Qurrat Ul Ain1, Jee Young Chung1, Yong-Hee Kim2.
Abstract
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.Entities:
Keywords: Engineered nucleases; Gene, protein and cell therapy; Genome silencing vs genome editing; Viral and non-viral delivery systems
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Year: 2014 PMID： 25553825 DOI： 10.1016/j.jconrel.2014.12.036
Source DB: PubMed Journal: J Control Release ISSN： 0168-3659 Impact factor: 9.776