Enhancing the Therapeutic Delivery of Oligonucleotides by Chemical Modification and Nanoparticle Encapsulation

Overview

Journal Molecules

Publisher MDPI

Specialty Biology

Date 2017 Oct 14

PMID 29027965

Citations 14

Authors

Yating Sun

Yarong Zhao

Xiuting Zhao

Robert J Lee

Lesheng Teng

Chenguang Zhou

Affiliations

Soon will be listed here.

Abstract

Oligonucleotide (ON) drugs, including small interfering RNA (siRNA), microRNA (miRNA) and antisense oligonucleotides, are promising therapeutic agents. However, their low membrane permeability and sensitivity to nucleases present challenges to in vivo delivery. Chemical modifications of the ON offer a potential solution to improve the stability and efficacy of ON drugs. Combined with nanoparticle encapsulation, delivery at the site of action and gene silencing activity of chemically modified ON drugs can be further enhanced. In the present review, several types of ON drugs, selection of chemical modification, and nanoparticle-based delivery systems to deliver these ON drugs are discussed.

Citing Articles

MicroRNA delivery based on nanoparticles of cardiovascular diseases.

Wang N, Chen C, Ren J, Dai D Mol Cell Biochem. 2023; 479(8):1909-1923.

PMID: 37542599 DOI: 10.1007/s11010-023-04821-0.

Emerging roles of noncoding RNAs in human cancers.

Wu S, Wu Y, Deng S, Lei X, Yang X Discov Oncol. 2023; 14(1):128.

PMID: 37439905 PMC: 10344860. DOI: 10.1007/s12672-023-00728-w.

Development and application of ribonucleic acid therapy strategies against COVID-19.

Ning L, Liu M, Gou Y, Yang Y, He B, Huang J Int J Biol Sci. 2022; 18(13):5070-5085.

PMID: 35982905 PMC: 9379410. DOI: 10.7150/ijbs.72706.

Nanodelivery of nucleic acids.

Mendes B, Conniot J, Avital A, Yao D, Jiang X, Zhou X Nat Rev Methods Primers. 2022; 2.

PMID: 35480987 PMC: 9038125. DOI: 10.1038/s43586-022-00104-y.

The development and improvement of ribonucleic acid therapy strategies.

Zhao Y, Shu R, Liu J Mol Ther Nucleic Acids. 2021; 26:997-1013.

PMID: 34540356 PMC: 8437697. DOI: 10.1016/j.omtn.2021.09.002.

References

Freire J, Rego de Figueiredo I, Valle J, Veiga A, Andreu D, Enguita F . siRNA-cell-penetrating peptides complexes as a combinatorial therapy against chronic myeloid leukemia using BV173 cell line as model. J Control Release. 2016; 245:127-136. DOI: 10.1016/j.jconrel.2016.11.027. View

Son S, Song S, Lee S, Min S, Kim S, Yhee J . Self-crosslinked human serum albumin nanocarriers for systemic delivery of polymerized siRNA to tumors. Biomaterials. 2013; 34(37):9475-85. DOI: 10.1016/j.biomaterials.2013.08.085. View

Liu H, Zhang Y, Xie Y, Cai Y, Li B, Li W . Hypoxia-responsive ionizable liposome delivery siRNA for glioma therapy. Int J Nanomedicine. 2017; 12:1065-1083. PMC: 5308568. DOI: 10.2147/IJN.S125286. View

Agrawal S, Temsamani J, Galbraith W, Tang J . Pharmacokinetics of antisense oligonucleotides. Clin Pharmacokinet. 1995; 28(1):7-16. DOI: 10.2165/00003088-199528010-00002. View

Simeoni F, Morris M, Heitz F, Divita G . Insight into the mechanism of the peptide-based gene delivery system MPG: implications for delivery of siRNA into mammalian cells. Nucleic Acids Res. 2003; 31(11):2717-24. PMC: 156720. DOI: 10.1093/nar/gkg385. View

Srikar R, Suresh D, Zambre A, Taylor K, Chapman S, Leevy M . Targeted nanoconjugate co-delivering siRNA and tyrosine kinase inhibitor to KRAS mutant NSCLC dissociates GAB1-SHP2 post oncogene knockdown. Sci Rep. 2016; 6:30245. PMC: 4987653. DOI: 10.1038/srep30245. View

Hsu C, Hendzel M, Uluda H . Improved transfection efficiency of an aliphatic lipid substituted 2 kDa polyethylenimine is attributed to enhanced nuclear association and uptake in rat bone marrow stromal cell. J Gene Med. 2011; 13(1):46-59. DOI: 10.1002/jgm.1526. View

Remaut K, Lucas B, Braeckmans K, Demeester J, De Smedt S . Pegylation of liposomes favours the endosomal degradation of the delivered phosphodiester oligonucleotides. J Control Release. 2006; 117(2):256-66. DOI: 10.1016/j.jconrel.2006.10.029. View

Abdelrahman M, Douziech Eyrolles L, Alkarib S, Herve-Aubert K, Ben Djemaa S, Marchais H . siRNA delivery system based on magnetic nanovectors: Characterization and stability evaluation. Eur J Pharm Sci. 2017; 106:287-293. DOI: 10.1016/j.ejps.2017.05.062. View

10.

Chang W, Wang Y, Zhu X, Wu C . Genome-Wide Profiling of miRNA and mRNA Expression in Alzheimer's Disease. Med Sci Monit. 2017; 23:2721-2731. PMC: 5467707. DOI: 10.12659/msm.905064. View

11.

Choong O, Mehrbod P, Tejo B, Omar A . In vitro antiviral activity of circular triple helix forming oligonucleotide RNA towards Feline Infectious Peritonitis virus replication. Biomed Res Int. 2014; 2014:654712. PMC: 3950953. DOI: 10.1155/2014/654712. View

12.

Li W, Chan C, Miller A, Lee C . Dual Functional Roles of Molecular Beacon as a MicroRNA Detector and Inhibitor. J Biol Chem. 2017; 292(9):3568-3580. PMC: 5339743. DOI: 10.1074/jbc.M116.765776. View

13.

Themis M, Forbes S, Chan L, Cooper R, Etheridge C, Miller A . Enhanced in vitro and in vivo gene delivery using cationic agent complexed retrovirus vectors. Gene Ther. 1999; 5(9):1180-6. DOI: 10.1038/sj.gt.3300715. View

14.

Aji Alex M, Nehate C, Veeranarayanan S, Kumar D, Kulshreshtha R, Koul V . Self assembled dual responsive micelles stabilized with protein for co-delivery of drug and siRNA in cancer therapy. Biomaterials. 2017; 133:94-106. DOI: 10.1016/j.biomaterials.2017.04.022. View

15.

Rassu G, Soddu E, Posadino A, Pintus G, Sarmento B, Giunchedi P . Nose-to-brain delivery of BACE1 siRNA loaded in solid lipid nanoparticles for Alzheimer's therapy. Colloids Surf B Biointerfaces. 2017; 152:296-301. DOI: 10.1016/j.colsurfb.2017.01.031. View

16.

Ragelle H, Colombo S, Pourcelle V, Vanvarenberg K, Vandermeulen G, Bouzin C . Intracellular siRNA delivery dynamics of integrin-targeted, PEGylated chitosan-poly(ethylene imine) hybrid nanoparticles: A mechanistic insight. J Control Release. 2015; 211:1-9. DOI: 10.1016/j.jconrel.2015.05.274. View

17.

Liu M, Li M, Sun S, Li B, Du D, Sun J . The use of antibody modified liposomes loaded with AMO-1 to deliver oligonucleotides to ischemic myocardium for arrhythmia therapy. Biomaterials. 2014; 35(11):3697-707. DOI: 10.1016/j.biomaterials.2013.12.099. View

18.

Antimisiaris S, Mourtas S, Papadia K . Targeted si-RNA with liposomes and exosomes (extracellular vesicles): How to unlock the potential. Int J Pharm. 2017; 525(2):293-312. DOI: 10.1016/j.ijpharm.2017.01.056. View

19.

Lam J, Armes S, Lewis A, Stolnik S . Folate conjugated phosphorylcholine-based polycations for specific targeting in nucleic acids delivery. J Drug Target. 2009; 17(7):512-23. DOI: 10.1080/10611860903023312. View

20.

Chen X, Dudgeon N, Shen L, Wang J . Chemical modification of gene silencing oligonucleotides for drug discovery and development. Drug Discov Today. 2005; 10(8):587-93. DOI: 10.1016/S1359-6446(05)03426-4. View