Intracellular Delivery of Antisense DNA and siRNA with Amino Groups Masked with Disulfide Units.
Chem Pharm Bull (Tokyo)
; 68(2): 129-132, 2020.
Article
de En
| MEDLINE
| ID: mdl-32009079
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.
Mots clés
Texte intégral:
1
Collection:
01-internacional
Base de données:
MEDLINE
Sujet principal:
ADN antisens
/
Petit ARN interférent
Limites:
Animals
/
Humans
/
Male
Langue:
En
Journal:
Chem Pharm Bull (Tokyo)
Année:
2020
Type de document:
Article