David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, Massachusetts 02139, USA.
Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, Massachusetts 02139, USA.
Nat Rev Drug Discov. 2017 Jun;16(6):387-399. doi: 10.1038/nrd.2016.280. Epub 2017 Mar 24.
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.
随着 CRISPR 技术的最新发展，对基因组进行工程改造变得越来越容易。基于 CRISPR 的基因组编辑系统，以及转录激活因子样效应物核酸酶（TALENs）和锌指核酸酶（ZFNs），正在成为生物医学研究、药物发现和开发，甚至基因治疗的有价值的工具。然而，对于这些系统中的每一个，为了有效地进入感兴趣的细胞并发挥其功能，都需要高效和安全的传递技术。这篇综述讨论了生物大分子传递和基因编辑的原理，考察了非病毒和病毒传递方法的最新进展和挑战，并强调了相关临床试验的现状。
