AI Article Synopsis
- Efficient delivery of antisense DNA and siRNA using cationic materials faces challenges like toxicity and poor effectiveness in reaching target cells.
- A bio-reactive mask that can be removed at the cell surface was developed to address these issues, avoiding harmful effects caused by exposed cationic groups.
- This innovative design allows for safer and more efficient delivery of oligonucleotides directly into the cell cytosol, showing potential for therapeutic use.
Article Abstract
Efficient methods for delivery of antisense DNA or small interfering RNA (siRNA) are highly needed. Cationic materials, which are conventionally used for anionic oligonucleotide delivery, have several drawbacks, including aggregate formation, cytotoxicity and a low endosome escape efficiency. In this report a bio-reactive mask (i.e., disulfide unit) for cationic amino groups was introduced, and the mask was designed such that it was removed at the target cell surface. Insolubility and severe cellular toxicity caused by exposed cationic groups are avoided when using the mask. Moreover, the disulfide unit used to mask the cationic group enabled direct delivery of oligonucleotides to the cell cytosol. The molecular design reported is a promising approach for therapeutic applications.
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| http://dx.doi.org/10.1248/cpb.c19-00811 | DOI Listing |
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