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Delivery technologies for genome editing
Nature Reviews Drug Discovery ( IF 120.1 ) Pub Date : 2017-03-24 00:00:00 , DOI: 10.1038/nrd.2016.280
Hao Yin , Kevin J. Kauffman , Daniel G. Anderson

With the recent development of CRISPR technology, it is becoming increasingly easy to engineer the genome. Genome-editing systems based on CRISPR, as well as transcription activator-like effector nucleases (TALENs) and zinc-finger nucleases (ZFNs), are becoming valuable tools for biomedical research, drug discovery and development, and even gene therapy. However, for each of these systems to effectively enter cells of interest and perform their function, efficient and safe delivery technologies are needed. This Review discusses the principles of biomacromolecule delivery and gene editing, examines recent advances and challenges in non-viral and viral delivery methods, and highlights the status of related clinical trials.

中文翻译:

基因组编辑的交付技术

随着CRISPR技术的最新发展,基因组工程变得越来越容易。基于CRISPR的基因组编辑系统以及类似转录激活子的效应核酸酶(TALEN)和锌指核酸酶(ZFN),正成为生物医学研究,药物发现和开发乃至基因治疗的重要工具。然而,为了使这些系统中的每一个有效进入感兴趣的细胞并执行其功能,需要有效且安全的递送技术。这篇综述讨论了生物大分子传递和基因编辑的原理,探讨了非病毒和病毒传递方法的最新进展和挑战,并重点介绍了相关临床试验的现状。
更新日期:2017-05-31
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