Gigante A, Li M, Junghänel S, Hirschhäuser C, Knauer S, Schmuck C
Institute of Organic Chemistry , University of Duisburg-Essen , 45141 Essen , Germany . Email:
Biomedical Technology Center of the Medical Faculty , University of Muenster , Muenster , Germany.
Medchemcomm. 2019 Aug 16;10(10):1692-1718. doi: 10.1039/c9md00275h. eCollection 2019 Oct 1.
Transfection is a process by which oligonucleotides (DNA or RNA) are delivered into living cells. This allows the synthesis of target proteins as well as their inhibition (gene silencing). However, oligonucleotides cannot cross the plasma membrane by themselves; therefore, efficient carriers are needed for successful gene delivery. Recombinant viruses are among the earliest described vectors. Unfortunately, they have severe drawbacks such as toxicity and immunogenicity. In this regard, the development of non-viral transfection vectors has attracted increasing interests, and has become an important field of research. In the first part of this review we start with a tutorial introduction into the biological backgrounds of gene transfection followed by the classical non-viral vectors (cationic organic carriers and inorganic nanoparticles). In the second part we highlight selected recent reports, which demonstrate that hybrid vectors that combine key features of classical carriers are a remarkable strategy to address the current challenges in gene delivery.
转染是一个将寡核苷酸（DNA或RNA）导入活细胞的过程。这使得能够合成靶蛋白并对其进行抑制（基因沉默）。然而，寡核苷酸自身无法穿过质膜；因此，为了成功进行基因递送，需要高效的载体。重组病毒是最早被描述的载体之一。不幸的是，它们存在诸如毒性和免疫原性等严重缺点。在这方面，非病毒转染载体的开发引起了越来越多的关注，并已成为一个重要的研究领域。在本综述的第一部分，我们首先对基因转染的生物学背景进行教程式介绍，随后介绍经典的非病毒载体（阳离子有机载体和无机纳米颗粒）。在第二部分，我们重点介绍一些近期的报告，这些报告表明，结合经典载体关键特征的杂合载体是应对当前基因递送挑战的一种卓越策略。
