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MicroRNAs as Biomarkers and Therapeutic Targets in Human Diseases

A special issue of International Journal of Molecular Sciences (ISSN 1422-0067). This special issue belongs to the section "Molecular Pathology, Diagnostics, and Therapeutics".

Deadline for manuscript submissions: 30 September 2025 | Viewed by 453

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School of Health Sciences, Faculty of Biology, Medicine and Health, University of Manchester, Oxford Rd., Manchester M13 9PT, UK
Interests: design and fabrication of supramolecular functional biomaterials with controlled chemical and biological properties by working at the interface between synthetic, analytical, physical and computational chemistry with structural and chemical biology
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Special Issue Information

Dear Colleagues,

Highly conserved endogenous, small non-coding microRNAs (miRNAs) regulate gene expression by modulating translational efficacy of their target mRNAs, thus affecting cellular functions, such as cell growth, division, differentiation, metabolism, neuronal gene expression, muscle differentiation and stem cell division. Distinctive profiles of miRNA expression are often associated with different pathological conditions ranging from neurodegenerative, cardiovascular and autoimmune diseases to cancer. The dysregulation in the miRNA expression may impact the normal functioning of cells, thus leading to development of various human diseases. Unique expression profiles of different miRNAs not only make them ideal biomarkers for diagnostic and prognostic purposes, but also offer the opportunity to explore this information for therapeutic purposes by exploiting them as biological targets or novel therapeutic tools.

In this Special Issue we are interested in all aspects of microRNA sequences that are relevant to (i) their applications in the diagnosis of human diseases, or to (ii) innovative therapeutic strategies, including (but not restricted to) therapeutic knockdown of pathogenic microRNAs, or miRNA mimetics for microRNA replacement therapy.

For this Special Issue, we invite manuscripts addressing one or several of the following aspects:

  1. Disease-related specific miRNAs expression patterns for early detection of human diseases
  2. Implication of miRNA in drug resistance
  3. Use of miRNAs as prognostic markers to predict patient’s response to therapy
  4. New strategies for fabrication of innovative diagnostics tools for robust, precise and reliable detection of microRNA expression profiles
  5. Therapeutic modulation of microRNAs using selective inhibition of miRNA function
  6. Use of microRNA replacement for therapeutics treatment
  7. Experimental evidence of substantial transformative potential of microRNA-based therapies

Dr. Elena V. Bichenkova
Guest Editor

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Keywords

  • microRNA biomarkers
  • diagnostic profiling
  • expression patterns
  • miRNA therapeutics
  • antimiRs
  • miRNA mimics
  • hybridisation
  • sequence-specificity
  • miRNA knockdown

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Published Papers (2 papers)

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Research

18 pages, 3497 KiB  
Article
Key Amniotic Fluid miRNAs as Promising Target Molecules for the Antenatal Prevention of Pulmonary Hypoplasia Associated with Congenital Diaphragmatic Hernia
by Angelika V. Timofeeva, Ivan S. Fedorov, Yuri I. Naberezhnev, Nana K. Tetruashvili and Gennady T. Sukhikh
Int. J. Mol. Sci. 2025, 26(8), 3872; https://doi.org/10.3390/ijms26083872 - 19 Apr 2025
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Abstract
Congenital diaphragmatic hernia (CDH) remains associated with high morbidity and mortality, primarily due to pulmonary hypoplasia and hypertension. Current antenatal diagnostic methods, such as ultrasound and magnetic resonance imaging (MRI), are unable to assess the severity of defects in lung and pulmonary vascular [...] Read more.
Congenital diaphragmatic hernia (CDH) remains associated with high morbidity and mortality, primarily due to pulmonary hypoplasia and hypertension. Current antenatal diagnostic methods, such as ultrasound and magnetic resonance imaging (MRI), are unable to assess the severity of defects in lung and pulmonary vascular structures, which are critical determinants of the diverse phenotypes of CDH. Aberrant epigenetic regulation of lung development during gestation is believed to play a significant role in the pathogenesis of CDH. In this study, we aimed to identify miRNA patterns in amniotic fluid capable of categorizing CDH-fetuses for the personalized selection of effective treatment strategies at the antenatal and/or postnatal stages. Using deep sequencing and quantitative real-time polymerase chain reaction (PCR), we identified a set of miRNAs—miR-485-3p, miR-320b, miR-320a-3p, miR-221-3p, miR-200b-3p, miR-100-5p, miR-92a-3p, miR-30c-5p, miR-26a-5p, and let-7c-5p—whose reduced expression in amniotic fluid at 19–24 weeks of gestation allowed us to categorize fetuses with CDH into two distinct groups: one significantly different from the control group (non-CDH) and the other closely resembling it. Notably, no significant correlations were found between the content of these miRNAs in amniotic fluid and severity of lung hypoplasia assessed by ultrasound or MRI. However, there was significant positive correlation between the level of each of the miRNAs with that of miR-200b-3p, whose role in ensuring proper bronchopulmonary tissue structure during prenatal development—as well as its therapeutic potential for CDH-associated hypoplastic lungs—has been previously demonstrated. These findings lay the groundwork for the future development of genetically engineered drug formulations designed for antenatal endotracheal administration to correct abnormal miRNA levels in lung tissue and mitigate the progression of pulmonary hypoplasia and hypertension in CDH-fetuses. Full article
(This article belongs to the Special Issue MicroRNAs as Biomarkers and Therapeutic Targets in Human Diseases)
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12 pages, 1071 KiB  
Article
Plasma Levels of MicroRNA Let-7c-5p May Predict Risk of Acute Chest Syndrome in Patients with Sickle Cell Disease
by James Fan, Joanna Gemel, Eric C. Beyer and Gabrielle Lapping-Carr
Int. J. Mol. Sci. 2025, 26(8), 3831; https://doi.org/10.3390/ijms26083831 - 18 Apr 2025
Viewed by 207
Abstract
Acute chest syndrome (ACS) is among the most serious complications of sickle cell disease (SCD). While the pathogenesis of ACS is incompletely understood, endothelial damage and microvascular occlusion are critical components. Our previous studies have implicated small extracellular vesicles in the plasma of [...] Read more.
Acute chest syndrome (ACS) is among the most serious complications of sickle cell disease (SCD). While the pathogenesis of ACS is incompletely understood, endothelial damage and microvascular occlusion are critical components. Our previous studies have implicated small extracellular vesicles in the plasma of subjects with SCD in causing endothelial dysfunction. This suggested that microRNAs within these small EVs might be responsible for endothelial damage. The sequencing of microRNAs in small EVs from the plasma of subjects with SCD revealed that several miRNAs were differentially expressed between subjects with and without ACS history, including let-7c-5p. In a replication cohort, plasma let-7c-5p levels were quantified via RT-qPCR. The baseline plasma let-7c-5p level was twofold higher in patients without previous ACS. Furthermore, we observed a positive correlation between let-7c-5p levels and time to subsequent ACS events. These findings suggest a role for let-7c-5p in endothelial disruption underlying ACS pathogenesis. It may also serve as a novel biomarker for ACS detection and the prediction of disease progression. Full article
(This article belongs to the Special Issue MicroRNAs as Biomarkers and Therapeutic Targets in Human Diseases)
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