Merkert Sylvia, Martin Ulrich
Leibniz Research Laboratories for Biotechnology and Artificial Organs (LEBAO), Department of Cardiothoracic, Transplantation and Vascular Surgery, Hannover Medical School, 30625 Hannover, Germany.
Regenerative Biology and Reconstructive Therapies (REBIRTH), Cluster of Excellence, Hannover Medical School, 30625 Hannover, Germany.
Int J Mol Sci. 2016 Jun 24;17(7):1000. doi: 10.3390/ijms17071000.
The possibility to generate patient-specific induced pluripotent stem cells (iPSCs) offers an unprecedented potential of applications in clinical therapy and medical research. Human iPSCs and their differentiated derivatives are tools for diseases modelling, drug discovery, safety pharmacology, and toxicology. Moreover, they allow for the engineering of bioartificial tissue and are promising candidates for cellular therapies. For many of these applications, the ability to genetically modify pluripotent stem cells (PSCs) is indispensable, but efficient site-specific and safe technologies for genetic engineering of PSCs were developed only recently. By now, customized engineered nucleases provide excellent tools for targeted genome editing, opening new perspectives for biomedical research and cellular therapies.
生成患者特异性诱导多能干细胞（iPSC）的可能性为临床治疗和医学研究带来了前所未有的应用潜力。人类iPSC及其分化衍生物是用于疾病建模、药物发现、安全药理学和毒理学的工具。此外，它们还可用于生物人工组织工程，是细胞治疗的有前途的候选者。对于许多这些应用而言，对多能干细胞（PSC）进行基因改造的能力是必不可少的，但高效的位点特异性和安全的PSC基因工程技术直到最近才得以开发。到目前为止，定制的工程核酸酶为靶向基因组编辑提供了出色的工具，为生物医学研究和细胞治疗开辟了新的前景。
