Haas Simone A, Dettmer Viviane, Cathomen Toni
Toni Cathomen, Ph.D., Institute for Cell and Gene Therapy, Medical Center - University of Freiburg, Hugstetter Str. 55, 79106 Freiburg, Germany, Phone: +49 761 270 34800, Fax: + 49 761 270 37900, E-Mail:
Hamostaseologie. 2017 Jan 31;37(1):45-52. doi: 10.5482/HAMO-16-09-0035. Epub 2017 Jan 10.
Targeted genome editing with designer nucleases, such as zinc finger nucleases, TALE nucleases, and CRISPR-Cas nucleases, has heralded a new era in gene therapy. Genetic disorders, which have not been amenable to conventional gene-addition-type gene therapy approaches, such as disorders with dominant inheritance or diseases caused by mutations in tightly regulated genes, can now be treated by precise genome surgery. Moreover, engineered nucleases enable novel genetic interventions to fight infectious diseases or to improve cancer immunotherapies. Here, we review the development of the different classes of programmable nucleases, discuss the challenges and improvements in translating gene editing into clinical use, and give an outlook on what applications can expect to enter the clinic in the near future.
使用诸如锌指核酸酶、转录激活因子样效应物核酸酶和CRISPR-Cas核酸酶等定制核酸酶进行靶向基因组编辑，开创了基因治疗的新纪元。传统的基因添加型基因治疗方法对某些遗传疾病无能为力，如具有显性遗传的疾病或由严格调控基因中的突变引起的疾病，而现在可以通过精确的基因组手术来治疗。此外，工程化核酸酶能够实现新的基因干预，以对抗传染病或改善癌症免疫疗法。在此，我们回顾了不同类型可编程核酸酶的发展，讨论了将基因编辑转化为临床应用所面临的挑战和改进，并展望了在不久的将来有望进入临床的应用。
