miRNA-based therapies: strategies and delivery platforms for oligonucleotide and non-oligonucleotide agents

被引:235
作者
Baumann, Volker [1 ]
Winkler, Johannes [1 ]
机构
[1] Univ Vienna, Dept Pharmaceut Chem, A-1090 Vienna, Austria
基金
奥地利科学基金会;
关键词
MICRORNA REPLACEMENT THERAPY; BIOACTIVE SMALL MOLECULES; CANCER STEM-CELLS; DOWN-REGULATION; RNA INTERFERENCE; IN-VIVO; RT-PCR; HEPATOCELLULAR-CARCINOMA; CIRCULATING MICRORNAS; CHEMICAL-MODIFICATION;
D O I
10.4155/fmc.14.116
中图分类号
R914 [药物化学];
学科分类号
100701 ;
摘要
The discovery of miRNAs as important regulatory agents for gene expression has expanded the therapeutic opportunities for oligonucleotides. In contrast to siRNA, miRNA-targeted therapy is able to influence not only a single gene, but entire cellular pathways or processes. It is possible to supplement downregulated or non-functional miRNAs by synthetic oligonucleotides, as well as alleviating effects caused by overexpression of malignant miRNAs through artificial antagonists, either oligonucleotides or small molecules. Chemical oligonucleotide modifications together with an efficient delivery system seem to be mandatory for successful therapeutic application. While miRNA-based therapy benefits from the decades of research spent on other therapeutic oligonucleotides, there are some specific challenges associated with miRNA therapy, mainly caused by the short target sequence. The current status and recent progress of miRNA-targeted therapeutics is described and future challenges and potential applications in treatment of cancer and viral infections are discussed.
引用
收藏
页码:1967 / 1984
页数:18
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