Therapeutic genome editing: prospects and challenges.
Therapeutic genome editing: prospects and challenges.
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基于可编程核酸酶的基因组编辑技术的最新进展显著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建立更准确的病理过程的细胞和动物模型，拓宽了我们阐明遗传学对疾病的贡献的能力。可编程核酸酶的一个特别诱人的应用是有可能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。在这里，我们讨论了目前在开发可编程核酸酶疗法方面的进展以及未来的前景和挑战。
Recent advances in the development of genome editing technologies based on programmable nucleases have significantly 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 towards developing programmable nuclease-based therapies as well as future prospects and challenges.