Cholesterol conjugated spermine as a delivery modality of antisense oligonucleotide.
- Author:
Yoon Kyung IM
1
;
Myung Su KIM
;
Hoon YOO
Author Information
1. Department of Pharmacology and Dental Therapeutics, School of Dentistry, Chosun University, Gwangju, 501-759, Korea. hoon_yoo@chosun.ac.kr
- Publication Type:Original Article
- Keywords:
cholesterol conjugated spermine;
antisense oligonucleotide;
delivery
- MeSH:
Blood Proteins;
Cholesterol*;
Genes, Reporter;
Introns;
Luciferases;
Oligonucleotides, Antisense;
Spermine*
- From:International Journal of Oral Biology
2013;38(4):155-160
- CountryRepublic of Korea
- Language:English
-
Abstract:
The major issue in the development of nucleic acid based therapeutics is the inefficient delivery of these agents into cells. We prepared cholesterol conjugated spermine and evaluated its usefulness as a delivery modality for antisense oligonucleotides in HeLa-Luc cells. A 2'-O-methyl antisense oligonucleotide sequence, designed to correct splicing at an aberrant intron inserted into a normal luciferase reporter gene, was used for complex formation with cholesterol conjugated spermine. Effective delivery of this antisense agent into nucleus would results in the expression of a luciferasereporter gene product. The cholesterol-spermine formed stable complexes with the antisense oligonucleotide and showed modest delivery activity. Furthermore, this delivery activity was maintained even in the presence of serum proteins, mimicking in vivo conditions. Cholesterol-spermine thus has potential as a delivery system for antisense oligonucleotides into cells.
