Trivalent Disulfide Unit-Masked System Efficiently Delivers Large Oligonucleotide.

ABSTRACT: Oligonucleotide drugs are shining in clinical therapeutics, but efficient and safe delivery systems severely limit their widespread use. A disulfide unit technology platform based on dynamic thiol exchange chemistry at the cell membrane has the potential for drug delivery. However, the alteration of the disulfide unit CSSC dihedral angle induced by different substituents directly affects the effectiveness of this technology and its stability. Previously, we constructed a trivalent low dihedral angle disulfide unit that can effectively promote the cellular uptake of small molecules. Here, we constructed a novel disulfide unit-masked oligonucleotide hybrid based on a low dihedral angle disulfide unit, motivated by prodrug design. Cellular imaging results showed that such a system exhibited s

SUBMITTER: Wang L 

PROVIDER: S-EPMC11397113 | biostudies-literature | 2024 Sep

REPOSITORIES: biostudies-literature