title: Current and future delivery systems for engineered nucleases: ZFN, TALEN and RGEN.
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.
authors.affiliation: Department of Bioengineering, Institute for Bioengineering and Biopharmaceutical Research, BK 21 Plus Future Biopharmaceutical Human Resources Training and Research Team, Hanyang University, 133-791 Seoul, Republic of Korea.
authors.forename: Qurrat
authors.initials: Q
authors.lastname: Ul Ain
authors.affiliation: Department of Bioengineering, Institute for Bioengineering and Biopharmaceutical Research, BK 21 Plus Future Biopharmaceutical Human Resources Training and Research Team, Hanyang University, 133-791 Seoul, Republic of Korea.
authors.forename: Jee Young
authors.initials: JY
authors.lastname: Chung
authors.affiliation: Department of Bioengineering, Institute for Bioengineering and Biopharmaceutical Research, BK 21 Plus Future Biopharmaceutical Human Resources Training and Research Team, Hanyang University, 133-791 Seoul, Republic of Korea. Electronic address: yongheekim@hanyang.ac.kr.
authors.forename: Yong-Hee
authors.initials: YH
authors.lastname: Kim
mesh_terms: D000818:Animals; D064130:CRISPR-Associated Proteins / Q000235:genetics; D064113:CRISPR-Cas Systems; D064112:Clustered Regularly Interspaced Short Palindromic Repeats / Q000235:genetics*; D005786:Gene Expression Regulation; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors; D006801:Humans; D015202:Protein Engineering / Q000379:methods*; D012260:Ribonucleases / Q000737:chemistry / Q000235:genetics* / Q000378:metabolism; D052416:Targeted Gene Repair; D014780:Viruses / Q000235:genetics; D016335:Zinc Fingers / Q000235:genetics*
chemical_list: D064130:CRISPR-Associated Proteins; D012260:Ribonucleases
keywords: Engineered nucleases; Gene, protein and cell therapy; Genome silencing vs genome editing; Viral and non-viral delivery systems