治疗性基因组编辑：前景与挑战。
Therapeutic genome editing: prospects and challenges.
作者信息
Cox David Benjamin Turitz, Platt Randall Jeffrey, Zhang Feng
机构信息
1] Broad Institute of Harvard and MIT, Cambridge, Massachusetts, USA. [2] Department of Health Sciences and Technology, Massachusetts Institute of Technology, Cambridge, Massachusetts, USA. [3] Department of Biology, Massachusetts Institute of Technology, Cambridge, Massachusetts, USA. [4] McGovern Institute for Brain Research at MIT, Cambridge, Massachusetts, USA.
1] Broad Institute of Harvard and MIT, Cambridge, Massachusetts, USA. [2] McGovern Institute for Brain Research at MIT, Cambridge, Massachusetts, USA. [3] Department of Brain and Cognitive Sciences, Massachusetts Institute of Technology, Cambridge, Massachusetts, USA. [4] Department of Biological Engineering, Massachusetts Institute of Technology, Cambridge, Massachusetts, USA.
出版信息
Nat Med. 2015 Feb;21(2):121-31. doi: 10.1038/nm.3793.
Recent advances in the development of genome editing technologies based on programmable nucleases have substantially improved our ability to make precise changes in the genomes of eukaryotic cells. Genome editing is already broadening our ability to elucidate the contribution of genetics to disease by facilitating the creation of more accurate cellular and animal models of pathological processes. A particularly tantalizing application of programmable nucleases is the potential to directly correct genetic mutations in affected tissues and cells to treat diseases that are refractory to traditional therapies. Here we discuss current progress toward developing programmable nuclease-based therapies as well as future prospects and challenges.
基于可编程核酸酶的基因组编辑技术的最新进展，极大地提高了我们在真核细胞基因组中进行精确改变的能力。基因组编辑已经通过促进创建更准确的病理过程细胞和动物模型，拓宽了我们阐明遗传学对疾病贡献的能力。可编程核酸酶一个特别诱人的应用是直接纠正受影响组织和细胞中的基因突变，以治疗传统疗法难以治愈的疾病。在此，我们讨论了开发基于可编程核酸酶疗法的当前进展以及未来前景和挑战。
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