| 摘要: |
| 锌指核酸酶技术是一种新兴的基因高效靶向修饰和调控技术，目前该技术已在人类疾病的基因治疗中得到应用，通过敲除致病基因使其丧失致病性或者通过修复突变基因使基因表达正常，实现了对多种人类疾病的基因治疗。基于提高锌指核酸酶效率、特异性和降低细胞毒性等方面的研究较少的现状，特就人工锌指核酸酶介导的基因敲除和基因同源重组修复等在人类疾病基因治疗方面的应用及存在问题进行了综述，并对相关研究的开展进行了展望。 |
| 关键词: 基因治疗 锌指核酸酶 同源重组 靶向修饰 |
| DOI： |
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| 基金项目:西北农林科技大学引进人才启动基金项目(2111020821) |
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| Progress on applications of zinc finger nuclease technology in gene therapy |
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| Abstract: |
| Zinc finger nuclease technology can be used effectively in gene regulation and modification. A number of recent papers have shown how this technology can be applied effectively to models of human gene therapy.A variety of human diseases have been treated by gene inactivation or gene correction.However,few papers focus on how to improve the efficiency of zinc finger nucleases and specificity,minimize cytotoxicity.The applications of this technology to human gene therapy and problems were summarized in this review to provide a basis for related researches. |
| Key words: gene therapy zinc finger nucleases homologous recombination targeting modification |
