Innovative Genomics Institute, University of California , Berkeley, California 94720, United States.
California Institute for Quantitative Biosciences, University of California , Berkeley, California 94720, United States.
ACS Chem Biol. 2018 Feb 16;13(2):376-382. doi: 10.1021/acschembio.7b00680. Epub 2017 Oct 19.
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.
基于 CRISPR 的基因组编辑技术有望为预防、治疗或治愈具有遗传基础的疾病带来无数新疗法。然而，安全有效地递送达基因组编辑酶是一个重大挑战，必须加以解决，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治疗遗传疾病的酶工具的最新进展，并探讨了将这些酶递送到需要纠正的细胞中的当前努力。已经用于传统基因治疗的病毒载体正被应用于实现体内基于 CRISPR 的治疗，而新兴技术，如基于纳米颗粒的 CRISPR 成分传递和预组装 RNA-蛋白复合物的直接传递，也正在被应用。在这些领域取得成功将使基于 CRISPR 的基因组编辑治疗发挥其全部潜力。
