National Laboratory of Biomacromolecules, Institute of Biophysics, Chinese Academy of Sciences, Beijing, 100101, China.
Protein Cell. 2011 Dec;2(12):950-6. doi: 10.1007/s13238-011-1132-0. Epub 2011 Dec 15.
Genetic manipulation of human pluripotent stem cells (hPSCs) provides a powerful tool for modeling diseases and developing future medicine. Recently a number of independent genome-editing techniques were developed, including plasmid, bacterial artificial chromosome, adeno-associated virus vector, zinc finger nuclease, transcription activator-like effecter nuclease, and helper-dependent adenoviral vector. Gene editing has been successfully employed in different aspects of stem cell research such as gene correction, mutation knock-in, and establishment of reporter cell lines (Raya et al., 2009; Howden et al., 2011; Li et al., 2011; Liu et al., 2011b; Papapetrou et al., 2011; Sebastiano et al., 2011; Soldner et al., 2011; Zou et al., 2011a). These techniques combined with the utility of hPSCs will significantly influence the area of regenerative medicine.
人类多能干细胞(hPSCs)的遗传操作提供了一种强大的工具,可用于疾病建模和未来医学的发展。最近开发了许多独立的基因组编辑技术,包括质粒、细菌人工染色体、腺相关病毒载体、锌指核酸酶、转录激活因子样效应核酸酶和辅助依赖性腺病毒载体。基因编辑已成功应用于干细胞研究的多个方面,如基因校正、突变敲入和报告细胞系的建立(Raya 等人,2009 年;Howden 等人,2011 年;Li 等人,2011 年;Liu 等人,2011b 年;Papapetrou 等人,2011 年;Sebastiano 等人,2011 年;Soldner 等人,2011 年;Zou 等人,2011a 年)。这些技术与 hPSCs 的实用性相结合,将极大地影响再生医学领域。