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Published on: December 26, 2015
MicroRNA silencing in primates: towards development of novel therapeutics
Andreas Petri1, Morten Lindow, Sakari Kauppinen
1Santaris Pharma, Kogle Allé 6, Hørsholm, Denmark and Wilhelm Johannsen Centre for Functional Genome Research, Department of Cellular and Molecular Medicine, University of Copenhagen, Blegdamsvej 3, Copenhagen, Denmark.
Abstract:
MicroRNAs (miRNA) comprise an abundant class of small noncoding RNAs that act as important posttranscriptional regulators of gene expression. Accumulating evidence showing that aberrantly expressed miRNAs play important roles in human cancers underscores them as potential targets for therapeutic intervention. Recent reports on efficient miRNA silencing in rodents and nonhuman primates using high-affinity targeting by chemically modified antisense oligonucleotides highlight the utility of such compounds in the development of miRNA-based cancer therapeutics.
Insights
Small noncoding RNAs called microRNAs (miRNAs) regulate genes and are implicated in cancer. Antisense oligonucleotides offer a promising strategy for developing miRNA-based cancer therapeutics.
Area of Science:
- Molecular Biology
- Genetics
- Oncology
Background:
- MicroRNAs (miRNAs) are small noncoding RNAs that regulate gene expression posttranscriptionally.
- Aberrant miRNA expression is increasingly recognized for its role in human cancers.
- miRNAs represent potential therapeutic targets for cancer treatment.
Purpose of the Study:
- To explore the therapeutic potential of targeting microRNAs in cancer.
- To highlight the utility of antisense oligonucleotides for miRNA silencing in cancer therapy.
Main Methods:
- Utilizing chemically modified antisense oligonucleotides.
- Investigating high-affinity targeting of microRNAs.
- Evaluating miRNA silencing in preclinical models (rodents and nonhuman primates).
Main Results:
- Demonstrated efficient silencing of microRNAs using modified antisense oligonucleotides.
- Showcased the effectiveness of this approach in animal models.
- Highlighted the potential for developing miRNA-based cancer therapeutics.
Conclusions:
- Chemically modified antisense oligonucleotides are effective tools for miRNA silencing.
- This approach holds significant promise for the development of novel miRNA-based cancer therapeutics.
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