用于体内递送基因组编辑机制的工程材料。
Engineered materials for in vivo delivery of genome-editing machinery.
作者信息
Tong Sheng, Moyo Buhle, Lee Ciaran M, Leong Kam, Bao Gang
机构信息
Department of Bioengineering, Rice University, Houston, TX, USA.
Department of Biomedical Engineering, Columbia University, New York, NY, USA.
出版信息
Nat Rev Mater. 2019 Nov;4:726-737. doi: 10.1038/s41578-019-0145-9. Epub 2019 Oct 4.
Genome editing technologies, such as CRISPR/Cas9, are promising for treating otherwise incurable genetic diseases. Great progress has been made for ex vivo genome editing; however, major bottlenecks exist in the development of efficient, safe, and targetable in vivo delivery systems, which are needed for the treatment of many diseases. To achieve high efficacy and safety in therapeutic in vivo genome editing, editing activities must be controlled spatially and temporally in the body, which requires novel materials, delivery strategies, and control mechanisms. Thus, there is currently a tremendous opportunity for the biomaterials research community to develop in vivo delivery systems that overcome the problems of low editing efficiency, off-targeting effect, safety, and cell and tissue specificity. In this Review, we summarize delivery approaches and provide perspectives on the challenges and possible solutions, aiming to stimulate further development of engineered materials for in vivo delivery of genome-editing machinery.
基因组编辑技术，如CRISPR/Cas9，有望用于治疗原本无法治愈的遗传疾病。体外基因组编辑已取得了巨大进展；然而，在开发高效、安全且可靶向的体内递送系统方面仍存在重大瓶颈，而许多疾病的治疗都需要这类系统。为了在体内治疗性基因组编辑中实现高疗效和安全性，编辑活性必须在体内进行时空控制，这需要新型材料、递送策略和控制机制。因此，目前生物材料研究领域有巨大的机会来开发体内递送系统，以克服编辑效率低、脱靶效应、安全性以及细胞和组织特异性等问题。在本综述中，我们总结了递送方法，并对挑战和可能的解决方案提出了看法，旨在推动用于体内递送基因组编辑工具的工程材料的进一步发展。
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