CRISPR-based genome editing technologies are poised to enable countless new therapies to prevent, treat, or cure diseases with a genetic basis. However, the safe and effective delivery of genome editing enzymes represents a substantial challenge that must be tackled to enable the next generation of genetic therapies. In this Review, we summarize recent progress in developing enzymatic tools to combat genetic disease and examine current efforts to deliver these enzymes to the cells in need of correction. Viral vectors already in use for traditional gene therapy are being applied to enable in vivo CRISPR-based therapeutics, as are emerging technologies such as nanoparticle-based delivery of CRISPR components and direct delivery of preassembled RNA–protein complexes. Success in these areas will allow CRISPR-based genome editing therapeutics to reach their full potential.

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基因组治疗的体内传递的承诺和挑战

基于CRISPR的基因组编辑技术已准备就绪，可以利用遗传学基础来实现无数新疗法来预防，治疗或治愈疾病。但是，安全有效地传递基因组编辑酶代表了一项重大挑战，必须进行应对才能实现下一代基因治疗。在这篇综述中，我们总结了开发用于对抗遗传疾病的酶学工具的最新进展，并研究了当前将这些酶传递给需要校正的细胞的努力。已经用于传统基因治疗的病毒载体正在应用于体内基于CRISPR的疗法以及新兴技术，例如基于纳米颗粒的CRISPR组分的递送和预组装RNA-蛋白复合物的直接递送。这些领域的成功将使基于CRISPR的基因组编辑疗法发挥其全部潜力。