Department of Biochemistry, University of Illinois at Urbana-Champaign, 600 South Mathews Avenue, Urbana, Illinois, 61801.
Biotechnol Bioeng. 2014 Apr;111(4):683-91. doi: 10.1002/bit.25154. Epub 2013 Dec 11.
Transcription activator-like effector nucleases (TALENs) have rapidly emerged as a powerful genome editing tool. The site-specific DNA double-strand breaks generated by TALENs in the human chromosome can induce homologous recombination or non-homologous end joining, resulting in desired genetic modifications. In this study, we report the development of a TALEN variant, SunnyTALEN, with >2.5-fold improved genome editing efficacy in human cells. The corresponding scaffold increases the rate of genetic modification at all the 13 tested loci of human genome and is compatible with heterodimer TALEN architectures. This enhanced and high-efficiency TALEN variant represents a novel second-generation TALEN system and has great potential for biological and therapeutic applications.
转录激活因子样效应物核酸酶(TALENs)已迅速成为一种强大的基因组编辑工具。TALEN 在人染色体上产生的靶向 DNA 双链断裂可诱导同源重组或非同源末端连接,从而导致所需的遗传修饰。在这项研究中,我们报告了一种 TALEN 变体 SunnyTALEN 的开发,该变体在人细胞中的基因组编辑效率提高了 >2.5 倍。相应的支架增加了人基因组中 13 个测试位点的遗传修饰率,并且与异源二聚体 TALEN 结构兼容。这种增强的高效 TALEN 变体代表了一种新型的第二代 TALEN 系统,在生物学和治疗应用方面具有巨大的潜力。