An Endosomal Escape Trojan Horse Platform to Improve Cytosolic Delivery of Nucleic Acids.

ABSTRACT: Endocytosis is a major bottleneck toward cytosolic delivery of nucleic acids, as the vast majority of nucleic acid drugs remain trapped within endosomes. Current trends to overcome endosomal entrapment and subsequent degradation provide varied success; however, active delivery agents such as cell-penetrating peptides have emerged as a prominent strategy to improve cytosolic delivery. Yet, these membrane-active agents have poor selectivity for endosomal membranes, leading to toxicity. A hallmark of endosomes is their acidic environment, which aids in degradation of foreign materials. Here, we develop a pH-triggered spherical nucleic acid that provides smart antisense oligonucleotide (ASO) release upon endosomal acidification and selective membrane disruption, termed DNA EndosomaL Escape Veh

SUBMITTER: Narum S 

PROVIDER: S-EPMC10906071 | biostudies-literature | 2024 Feb

REPOSITORIES: biostudies-literature