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Advances in Regulatory Mechanisms and Therapeutic Potential of Non-Coding RNAs...
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Advances in Regulatory Mechanisms and Therapeutic Potential of Non-Coding RNAs in Neurodegenerative Diseases

A special issue of Pharmaceuticals (ISSN 1424-8247). This special issue belongs to the section "Biopharmaceuticals".

Deadline for manuscript submissions: 30 June 2026 | Viewed by 3193

Special Issue Editors

Dr. Paola Ruffo

E-Mail Website
Guest Editor
Neuromuscular Diseases Research Section, National Institute on Aging, National Institutes of Health, Bethesda, MD 20892, USA
Interests: amyotrophic lateral sclerosis; non-coding RNA; computational biology
Dr. Maurizio Grassano

E-Mail Website
Guest Editor
‘Rita Levi Montalcini’ Department of Neuroscience, ALS Center, University of Turin, Turin, Italy
Interests: neurology; neurodegeneration; amyotrophic lateral sclerosis

Special Issue Information

Dear Colleagues,

Neurodegenerative diseases—such as amyotrophic lateral sclerosis (ALS), Alzheimer’s disease (AD), and Parkinson’s disease (PD) —are complex and multifactorial disorders characterized by progressive neuronal dysfunction and loss. Accumulating evidence underscores the critical roles of non-coding RNAs (ncRNAs), including microRNAs, long non-coding RNAs, and circular RNAs, in modulating gene expression, synaptic plasticity, mitochondrial function, and neuroinflammatory pathways implicated in disease pathogenesis. 

This Special Issue will spotlight recent advances in elucidating the molecular mechanisms through which ncRNAs contribute to neurodegeneration. It will also highlight their translational potential as biomarkers for early diagnosis and disease progression and their promise as therapeutic targets in precision medicine. We welcome original research articles and reviews focusing on the mechanistic insights into ncRNA biology, novel regulatory networks, and the preclinical or clinical applications of ncRNA-based strategies.

Additionally, this collection aims to foster a comprehensive understanding of ncRNA functions in the central nervous system and catalyze the development of innovative diagnostic and therapeutic approaches for these devastating diseases by integrating findings from experimental and clinical research.

Dr. Paola Ruffo
Dr. Maurizio Grassano
Guest Editors

Manuscript Submission Information

Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All submissions that pass pre-check are peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 250 words) can be sent to the Editorial Office for assessment.

Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. Pharmaceuticals is an international peer-reviewed open access monthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 2900 CHF (Swiss Francs). Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.

Keywords

  • non-coding RNAs
  • microRNAs
  • long non-coding RNAs
  • circular RNAs
  • neurodegeneration
  • Alzheimer's disease
  • Parkinson's disease
  • amyotrophic lateral sclerosis
  • gene regulation
  • biomarkers
  • RNA therapeutics
  • neuroinflammation
  • synaptic dysfunction
  • translational neuroscience

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Published Papers (2 papers)

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Review

39 pages, 3332 KB  
Review
The Expanding Role of Non-Coding RNAs in Neurodegenerative Diseases: From Biomarkers to Therapeutic Targets
by Xuezhi Zhao, Yongquan Zheng, Xiaoyu Cai, Yao Yao and Dongxu Qin
Pharmaceuticals 2026, 19(1), 92; https://doi.org/10.3390/ph19010092 - 3 Jan 2026
Viewed by 1506
Abstract
Non-coding RNAs have emerged as central regulators of gene expression in neurodegenerative diseases, offering new opportunities for diagnosis and therapy. This review synthesizes current knowledge on microRNAs, long non-coding RNAs, and circular RNAs in Alzheimer’s disease, Parkinson’s disease, and amyotrophic lateral sclerosis, emphasizing [...] Read more.
Non-coding RNAs have emerged as central regulators of gene expression in neurodegenerative diseases, offering new opportunities for diagnosis and therapy. This review synthesizes current knowledge on microRNAs, long non-coding RNAs, and circular RNAs in Alzheimer’s disease, Parkinson’s disease, and amyotrophic lateral sclerosis, emphasizing their roles in synaptic function, proteostasis, mitochondrial biology, and neuroinflammation. We evaluate evidence supporting non-coding RNAs as circulating and tissue-based biomarkers for early detection, disease monitoring, and patient stratification, and we compare analytical platforms and biofluid sources. Mechanistic insights reveal how non-coding RNAs modulate pathogenic protein aggregation, neuronal excitability, immune cell crosstalk, and blood–brain barrier integrity. Translational efforts toward RNA-targeted interventions are reviewed, including antisense oligonucleotides, small interfering RNAs, miRNA mimics and inhibitors, circular RNA decoys, and extracellular vesicle-mediated delivery systems. We discuss pharmacological modulation, delivery challenges, safety concerns, and strategies to enhance specificity and CNS penetration. Finally, we outline emerging computational and multi-omics approaches to prioritize therapeutic targets and propose a roadmap for advancing non-coding RNA research from preclinical models to clinical trials. Addressing biological heterogeneity and delivery barriers will be pivotal to realizing the diagnostic and therapeutic promise of the non-coding transcriptome in neurodegenerative disease. Collaboration across disciplines and rigorous clinical validation are urgently needed. Full article
(This article belongs to the Special Issue Advances in Regulatory Mechanisms and Therapeutic Potential of Non-Coding RNAs in Neurodegenerative Diseases)
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19 pages, 788 KB  
Review
The Other Side of the Same Coin: Beyond the Coding Region in Amyotrophic Lateral Sclerosis
by Paola Ruffo, Benedetta Perrone, Francesco Perrone, Francesca De Amicis, Rodolfo Iuliano, Cecilia Bucci, Angela Messina and Francesca Luisa Conforti
Pharmaceuticals 2025, 18(10), 1573; https://doi.org/10.3390/ph18101573 - 18 Oct 2025
Viewed by 1334
Abstract
Transposable elements (TEs), once regarded as genomic “junk,” are now recognized as powerful regulators of gene expression, genome stability, and innate immunity. In the context of neurodegeneration, particularly Amyotrophic Lateral Sclerosis (ALS), accumulating evidence implicates TEs as active contributors to disease pathogenesis. ALS [...] Read more.
Transposable elements (TEs), once regarded as genomic “junk,” are now recognized as powerful regulators of gene expression, genome stability, and innate immunity. In the context of neurodegeneration, particularly Amyotrophic Lateral Sclerosis (ALS), accumulating evidence implicates TEs as active contributors to disease pathogenesis. ALS is a fatal motor neuron disease with both sporadic and familial forms, linked to genetic, epigenetic, and environmental factors. While coding mutations explain a subset of cases, advances in long-read sequencing and epigenomic profiling have unveiled the profound influence of non-coding regions—especially retrotransposons such as LINE-1, Alu, and SVA—on ALS onset and progression. TEs may act through multiple mechanisms: generating somatic mutations, disrupting chromatin architecture, modulating transcriptional networks, and triggering sterile inflammation via innate immune pathways like cGAS-STING. Their activity is normally repressed by epigenetic regulators, including DNA methylation, histone modifications, and RNA interference pathways; however, these controls are compromised in ALS. Taken together, these insights underscore the translational potential of targeting transposable elements in ALS, both as a source of novel biomarkers for patient stratification and disease monitoring, and as therapeutic targets whose modulation may slow neurodegeneration and inflammation. This review synthesizes the current knowledge of TE biology in ALS; integrates findings across molecular, cellular, and systems levels; and explores the therapeutic potential of targeting TEs as modulators of neurodegeneration. Full article
(This article belongs to the Special Issue Advances in Regulatory Mechanisms and Therapeutic Potential of Non-Coding RNAs in Neurodegenerative Diseases)
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