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.
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.
J Control Release. 2015 May 10;205:120-7. doi: 10.1016/j.jconrel.2014.12.036. Epub 2014 Dec 30.
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.
基因治疗是一种遗传干预方法,通过针对特定基因的特定核苷酸进行工程化核酸酶的设计,以建立、抑制、消除功能或纠正突变来治疗遗传性疾病。在这里,我们综述了利用靶向基因组工程的快速发展技术,包括锌指核酸酶(ZFNs)、转录激活因子样核酸酶(TALENs)和基于簇状规则间隔短回文重复序列/CRISPR 相关蛋白(CRISPR/Cas)的 RNA 指导的 DNA 内切酶(RGENs),及其不同的特性,包括这些核酸酶进行基因组修饰的优缺点。我们还进一步讨论了诱导基因编辑的不同类型的递送方法、除核酸酶以外的基因工程的新进展以及未来的前景。