Emerging Concepts in miRNA-Based Therapeutics

A special issue of Genes (ISSN 2073-4425). This special issue belongs to the section "Molecular Genetics and Genomics".

Deadline for manuscript submissions: closed (15 August 2024) | Viewed by 1773

Special Issue Editor


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Guest Editor
Department of Physiology and Cellular Biophysics, Columbia University, 1150 St Nicholas Ave, Russ Berrie Science Pavilion 515, New York, NY 10032, USA
Interests: microRNA; calcium channels; cardiovascular physiology and disease

Special Issue Information

Dear Colleagues,

microRNAs (miRNAs, miRs) are short (18–26 nucleotides) regulatory RNA molecules. They were discovered in 1993 in Caenorhabditis elegans (C. elegans). Since then, there has been an exponential growth in the number of publications describing new members of this emergent nucleic acid family, elucidating their biogenesis, mechanisms of action, and their roles in diverse signaling pathways and physiological processes, as well as their contribution to the development of multiple pathologies.

miRNAs act mainly by downregulating genes to whose mRNAs they are (partially) complementary. As almost ubiquitous master regulators of crucially important cellular functions and physiological processes, in recent years, miRNAs have become new promising diagnostic tools and therapeutic targets in multiple pathological conditions, from asthma and cancer, through cardiovascular and metabolic diseases, to Parkinson’s disease and Zika virus infection.

Some therapeutic applications reached clinical trial stages, but in spite of great progress in basic research and preclinical studies and the development of stable and effective delivery vehicles, as of today, no miRNA-based therapeutic has been approved for treatment. However, there is a consensus in the gene therapy field that this is just a matter of time.

Some of the main obstacles in fulfilling miRNAs’ promise as therapeutic agents are the wide range of downstream effects and safe and effective targeted delivery.

This Special Issue aims at presenting the most recent advancements in miRNA-based therapeutics, including new miRNA targets and novel promising approaches and solutions to overcoming existing obstacles in therapeutic applications. It also invites new perspectives and paradigms that might lead to a breakthrough in the field, hopefully leading to the development of the first approved miRNA-based therapeutic agents.

Dr. Anetta Wronska
Guest Editor

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Keywords

  • microRNA
  • miRNA
  • miR
  • gene therapy
  • non-coding RNA
  • microRNA-based therapeutics

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Published Papers (1 paper)

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Research

13 pages, 2409 KiB  
Article
Systematic Analysis of miR-506-3p Target Genes Identified Key Mediators of Its Differentiation-Inducing Function
by Daniela F. Cardus, Mitchell T. Smith, Alexandra Vernaza, Jadyn L. Smith, Brynn Del Buono, Anupa Parajuli, Emma G. Lewis, Nakya Mesa-Diaz and Liqin Du
Genes 2024, 15(10), 1268; https://doi.org/10.3390/genes15101268 - 27 Sep 2024
Cited by 1 | Viewed by 1347
Abstract
Background/Objectives: miR-506-3p has been demonstrated to be a strong inducer of neuroblastoma cell differentiation, highlighting the potential of applying miR-506-3p mimics to neuroblastoma differentiation therapy. However, the target genes of miR-506-3p that mediate its differentiation-inducing function have not been fully defined. This [...] Read more.
Background/Objectives: miR-506-3p has been demonstrated to be a strong inducer of neuroblastoma cell differentiation, highlighting the potential of applying miR-506-3p mimics to neuroblastoma differentiation therapy. However, the target genes of miR-506-3p that mediate its differentiation-inducing function have not been fully defined. This study aims to comprehensively investigate the targetome of miR-506-3p regarding its role in regulating neuroblastoma cell differentiation. Methods: We combined gene expression profiling and functional high-content screening (HCS) to identify miR-506-3p target genes that have differentiation-modulating functions. For evaluating the potential clinical relevance of the identified genes, we analyzed the correlations of gene expressions with neuroblastoma patient survival. Results: We identified a group of 19 target genes with their knockdown significantly inducing cell differentiation, suggesting that these genes play a key role in mediating the differentiation-inducing activity of miR-506-3p. We observed significant correlations of higher mRNA levels with lower patient survival with 13 of the 19 genes, suggesting that overexpression of these 13 genes plays important roles in promoting neuroblastoma development by disrupting the cell differentiation pathways. Conclusions: Through this study, we identified novel target genes of miR-506-3p that function as strong modulators of neuroblastoma cell differentiation. Our findings represent a significant advancement in understanding the mechanisms by which miR-506-3p induces neuroblastoma cell differentiation. Future investigations of the identified 13 genes are needed to fully define their functions and mechanisms in controlling neuroblastoma cell differentiation, the understanding of which may reveal additional targets for developing novel differentiation therapeutic agents. Full article
(This article belongs to the Special Issue Emerging Concepts in miRNA-Based Therapeutics)
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