发育医学电子杂志  2018, Vol. 6 Issue (3): 134-140 DOI: 10.3969/j.issn.2095-5340.2018.03.002 |
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发育基础
述评
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| 基因编辑技术最新研究进展及在疾病治疗中的应用 |
| 项光海 王皓毅 |
| 1. 中国科学院动物研究所 干细胞与生殖生物学国家重点实验室，北京 100101；2. 中国科学院大学 存济医学院，北京 100049 |
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| Research advances and therapeutic applications of gene editing |
| XIANG Guang-hai , WANG Hao-yi |
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摘要 基因编辑技术是指用可编程的核酸酶识别基因组特定位点并介导 DNA 双链断裂，随后诱发DNA 非同源末端连接（non-homology end-joining，NHEJ）或同源重组修复（homology directed repair，HDR）等机制，从而实现对 DNA 序列的定点修饰，包括靶向敲除或敲入基因。20 世纪 90 年代以来，科学家开发了一系列基因编辑工具，包括 Meganuclease、锌 指 核 酸 酶（zinc-?nger nucleases，ZFN）、转 录 激 活因 子 样 效 应 物 核 酸 酶（transcription activator-like effector nucleases，TALEN）等，能够高效地特异性造成基因组目标位点双链 DNA 断裂，提高同源重组的效率［1-2］。近年来建立的成簇规律间隔短回文重复序列（clustered regularly interspaced short palindromic repeat，CRISPR）/Cas 系 统，具 有 效 率高、操作简单、易于改造优化等优势，目前已经成为最重要的基因编辑工具，并被广泛应用于基因表达调控、细胞成像、核酸标记、表观遗传修饰、疾病治疗、功能基因筛选等领域。本文主要阐述 CRISPR/Cas9 技术优化方面以及在生物检测和疾病治疗等领域的研究进展。
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| 关键词:
CRISPR/Cas9 技术')" href="#">CRISPR/Cas9 技术
细胞治疗技术')" href="#"> 细胞治疗技术
基因编辑技术')" href="#"> 基因编辑技术
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收稿日期: 2018-06-26
出版日期: 2018-07-30
发布日期: 2018-08-20
期的出版日期: 2018-07-30
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| 基金资助: 科技部 863 计划（2015AA020307）；中国科学院战略性先导科技计划（XDA16010205）；国家自然科学基金（31471215） |
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通讯作者:
王皓毅https://baike.baidu.com/item/%E7%8E%8B%E7%9A%93%E6%AF%85
E-mail: wanghaoyi@ioz.ac.cn
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| [1] |
GAO Xiao-hui, MAO Jian. Clinical features of non-oliguric hyperkalemia in extremely low birth weight infants[J]. Journal of Developmental Medicine(Electronic Version), 0, (): 152
. |
| [2] |
Society of Neonatologist, Chinese Medical Doctor Association. Consensus recommendations on the prevention and early management of respiratory distress syndrome in preterm infants[J]. Journal of Developmental Medicine(Electronic Version), 2017, 5(3): 129
-131
. |
| [3] |
Professional Committee of Respiratory, Society of Neonatologist, Chinese Medical Doctor Association. Clinical application recommendations for heated humidified high flow nasal cannula[J]. Journal of Developmental Medicine(Electronic Version), 2017, 5(3): 132
-135
. |
| [4] |
YAN Jun, ZHU Xing-wang, SHI Yuan. Application progress of noninvasive ventilate technique for premature infants[J]. Journal of Developmental Medicine(Electronic Version), 2017, 5(3): 136
-140
. |
| [5] |
GU Min-fang, YANG Chuan-zhong. Progress of intrapartum resuscitation for premature infants[J]. Journal of Developmental Medicine(Electronic Version), 2017, 5(3): 141
-145
. |
| [6] |
LIU Shu-hua, SHEN Yue-bo, LIU Cui-qing, MA Li. The efficacy of pulmonary surfactant for pulmonary function in premature tension pneumothorax[J]. Journal of Developmental Medicine(Electronic Version), 2017, 5(3): 146
-151
. |
| [7] |
GAO Xiao-hui, MAO Jian. Clinical features of non-oliguric hyperkalemia in extremely low birth weight infants[J]. Journal of Developmental Medicine(Electronic Version), 2017, 5(3): 152
-158
. |
| [8] |
XIA Yao-fang, YANG Juan , TIAN Bao-li, et al. Value of amplitude-integrated electroencephalography in monitoring acute period of neonatal bilirubin encephalopathy and prognostic assessment[J]. Journal of Developmental Medicine(Electronic Version), 2017, 5(3): 159
-163
. |
| [9] |
WANG Li-rong, SUN Xiao-yan, ZHU Ruo-xin, et al. Epidemiological investigation and analysis of women aged 40-55 years old with osteoporosis in Gansu province[J]. Journal of Developmental Medicine(Electronic Version), 2017, 5(3): 164
-167
. |
| [10] |
CHEN Ru-yue, SHEN Yun-yan, CHEN Qing , et al. Five cases about Henoch-Schönlein purpura complicated with central nervous system injury in children and literatures review[J]. Journal of Developmental Medicine(Electronic Version), 2017, 5(3): 168
-171
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