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Cholesterol conjugated spermine as a delivery modality of antisense oligonucleotide KCI 등재후보

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대한구강생물학회 (The Korean Academy of Oral Biology)
초록

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.

저자
  • Yoon Kyung Im(Department of Pharmacology and Dental Therapeutics, School of Dentistry, Chosun University)
  • Myung Su Kim(Department of Pharmacology and Dental Therapeutics, School of Dentistry, Chosun University)
  • Hoon Yoo(Department of Pharmacology and Dental Therapeutics, School of Dentistry, Chosun University) Correspondence