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Strategies to use microRNAs as therapeutic targets


Krützfeldt, Jan (2016). Strategies to use microRNAs as therapeutic targets. Best Practice & Research. Clinical Endocrinology & Metabolism, 30(5):551-561.

Abstract

MicroRNAs (miRNAs) provide a unique mechanism of gene regulation and play a key role in different pathologies ranging from metabolic diseases to cancer. miRNAs can impact biological function as either suppressors of gene expression when their expression levels are enhanced in a disease state or they can cause upregulation of gene expression when their expression levels are reduced. Therefore both gain- and loss-of- function strategies are needed to fully exploit their therapeutic potential. miRNA research first focused on inhibition of single miRNAs using oligonucleotide inhibitors. However, more recent approaches explore the potential to deliver oligonucleotides to mimic miRNA expression or to employ small molecules to increase or inhibit miRNA function. Although we need to know more about the potential side effects and tissue specific delivery systems, these studies provide grounds to further exploit miRNAs as novel therapeutic targets in the clinic.

Abstract

MicroRNAs (miRNAs) provide a unique mechanism of gene regulation and play a key role in different pathologies ranging from metabolic diseases to cancer. miRNAs can impact biological function as either suppressors of gene expression when their expression levels are enhanced in a disease state or they can cause upregulation of gene expression when their expression levels are reduced. Therefore both gain- and loss-of- function strategies are needed to fully exploit their therapeutic potential. miRNA research first focused on inhibition of single miRNAs using oligonucleotide inhibitors. However, more recent approaches explore the potential to deliver oligonucleotides to mimic miRNA expression or to employ small molecules to increase or inhibit miRNA function. Although we need to know more about the potential side effects and tissue specific delivery systems, these studies provide grounds to further exploit miRNAs as novel therapeutic targets in the clinic.

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Additional indexing

Item Type:Journal Article, refereed, original work
Communities & Collections:04 Faculty of Medicine > University Hospital Zurich > Clinic for Endocrinology and Diabetology
Dewey Decimal Classification:610 Medicine & health
Language:English
Date:October 2016
Deposited On:16 Jan 2017 08:00
Last Modified:17 Jan 2017 08:44
Publisher:Elsevier
ISSN:1521-690X
Publisher DOI:https://doi.org/10.1016/j.beem.2016.07.004
PubMed ID:27923450

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