Engineered materials for in vivo delivery of genome-editing machinery.
Engineered materials for in vivo delivery of genome-editing machinery.
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DOI：
10.1038/s41578-019-0145-9
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发表时间：
2019-11
期刊：
影响因子：
--
通讯作者：
Bao G
中科院分区：
文献类型：
--
作者：
Tong S;Moyo B;Lee CM;Leong K;Bao G
基因组编辑技术，如CRISPR/Cas9，有望用于治疗其他无法治愈的遗传疾病。离体基因组编辑已经取得了很大进展;然而，在开发治疗许多疾病所需的有效、安全和可靶向的体内递送系统方面存在主要瓶颈。为了在治疗性体内基因组编辑中实现高效率和安全性，编辑活动必须在体内空间和时间上受到控制，这需要新的材料、递送策略和控制机制。因此，目前生物材料研究界有巨大的机会来开发克服低编辑效率、脱靶效应、安全性以及细胞和组织特异性的问题的体内递送系统。在这篇综述中，我们总结了交付方法，并提供了挑战和可能的解决方案的观点，旨在刺激进一步开发用于体内交付基因组编辑机器的工程材料。体内基因组编辑需要高效、安全且具有组织特异性的递送系统。本文概述了目前使用的材料和递送策略，以及体内基因组编辑中的挑战和潜在解决方案，旨在刺激进一步开发用于体内递送基因组编辑机器的工程材料。
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. In vivo genome editing requires delivery systems that are efficiency, safe, and have tissue specificity. This Review outlines the materials and delivery strategies currently used, and the challenges and potential solutions in in vivo genome editing, aiming to stimulate further development of engineered materials for in vivo delivery of genome-editing machinery.
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DOI：
10.1126/science.aaf5573
发表时间：
2016-08-05
期刊：
Science (New York, N.Y.)
影响因子：
--
作者：
Abudayyeh OO;Gootenberg JS;Konermann S;Joung J;Slaymaker IM;Cox DB;Shmakov S;Makarova KS;Semenova E;Minakhin L;Severinov K;Regev A;Lander ES;Koonin EV;Zhang F
通讯作者：
Zhang F
DOI：
10.1038/mt.2015.164
发表时间：
2016-03
期刊：
Molecular therapy : the journal of the American Society of Gene Therapy
影响因子：
--
作者：
Chen X;Gonçalves MA
通讯作者：
Gonçalves MA
影响因子：
82.9
作者：
Charlesworth, Carsten T.;Deshpande, Priyanka S.;Porteus, Matthew H.
通讯作者：
Porteus, Matthew H.
影响因子：
10.3
作者：
Dreher, MR;Liu, WG;Chilkoti, A
通讯作者：
Chilkoti, A
影响因子：
48
作者：
Dai, Xiaoyun;Park, Jonathan J.;Chen, Sidi
通讯作者：
Chen, Sidi