Pharmaceutical Biotechnology, Department of Pharmacy, Ludwig-Maximilians-Universität Munich, Butenandtstrasse 5-13, 81377 Munich, Germany; CNATM - Cluster for Nucleic Acid Therapeutics Munich, Germany.
Pharmaceutical Biotechnology, Department of Pharmacy, Ludwig-Maximilians-Universität Munich, Butenandtstrasse 5-13, 81377 Munich, Germany; CNATM - Cluster for Nucleic Acid Therapeutics Munich, Germany; Center for Nanoscience (CeNS), LMU Munich, 80799 Munich, Germany.
Curr Opin Chem Biol. 2024 Aug;81:102506. doi: 10.1016/j.cbpa.2024.102506. Epub 2024 Aug 2.
Despite impressive recent establishment of therapeutic nucleic acids as drugs and vaccines, their broader medical use is impaired by modest performance in intracellular delivery. Inefficient endosomal escape presents a major limitation responsible for inadequate cytosolic cargo release. Depending on the carrier, this endosomal barrier can strongly limit or even abolish nucleic acid delivery. Different recent endosomal escape strategies and their hypothesized mechanisms are reviewed.
尽管最近在治疗性核酸作为药物和疫苗的应用方面取得了令人瞩目的进展，但由于细胞内递送效果不佳，它们的广泛医学应用受到了限制。低效的内涵体逃逸是导致胞质内货物释放不足的主要限制因素。根据载体的不同，这种内涵体屏障可能会强烈限制甚至完全阻止核酸的递送。本文回顾了不同的近期内涵体逃逸策略及其假设的机制。
