Pharmaceutical Biotechnology, Department of Pharmacy and Center for Nanoscience (CeNS), Ludwig-Maximilians-University, Munich, Germany.
Nanomedicine (Lond). 2014 Dec;9(18):2843-59. doi: 10.2217/nnm.14.166.
Short synthetic oligonucleotides (ONs) are a group of therapeutic molecules with enormous clinical potential owing to their high specificity and ability to target the expression of virtually any single or group of genes. Clinical translation of ONs is hampered by the inadequate bioavailability in the target cells due to the substantial extracellular and intracellular barriers exposed to these molecules. Different cationic polymers have been successfully deployed for the delivery of ONs. However, heterogeneous nature of these classical polymers is not suitable for clinical applications and hence vectors with completely defined structure are required. In this review, we discuss recent advances with sequence-defined polymers and their application for the delivery of short ONs.
短链合成寡核苷酸 (ONs) 是一类具有巨大临床潜力的治疗分子，因为它们具有高度的特异性，并且能够靶向几乎任何单个或一组基因的表达。由于这些分子暴露于大量的细胞外和细胞内屏障，因此 ONs 在靶细胞中的生物利用度不足，这阻碍了它们的临床转化。不同的阳离子聚合物已成功用于 ONs 的递送。然而，这些经典聚合物的异质性不适合临床应用，因此需要具有完全定义结构的载体。在这篇综述中，我们讨论了序列定义聚合物的最新进展及其在短链 ONs 传递中的应用。
