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Targeting Non-Coding RNAs as a Potential Therapeutic and Delivery Strategy Against Neurodegenerative Diseases.

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  • Author(s): Bougea A;Bougea A
  • Source:
    International journal of molecular sciences [Int J Mol Sci] 2026 Apr 03; Vol. 27 (7). Date of Electronic Publication: 2026 Apr 03.
  • Publication Type:
    Journal Article; Review
  • Language:
    English
  • Additional Information
    • Source:
      Publisher: MDPI Country of Publication: Switzerland NLM ID: 101092791 Publication Model: Electronic Cited Medium: Internet ISSN: 1422-0067 (Electronic) Linking ISSN: 14220067 NLM ISO Abbreviation: Int J Mol Sci Subsets: MEDLINE
    • Publication Information:
      Original Publication: Basel, Switzerland : MDPI, [2000-
    • Subject Terms:
      Neurodegenerative Diseases*/genetics ; Neurodegenerative Diseases*/therapy ; RNA, Untranslated*/genetics; MicroRNAs/genetics ; RNA, Long Noncoding/genetics ; Genetic Therapy/methods ; Humans ; Animals
    • Abstract:
      Neurodegenerative diseases (NDs), including Alzheimer's disease, Parkinson's disease, Huntington's disease, and amyotrophic lateral sclerosis (ALS), represent a growing global health challenge characterized by progressive neuronal loss and a lack of definitive disease-modifying treatments. This review explores the emerging potential of targeting non-coding RNAs (ncRNAs), such as microRNAs (miRNAs), long non-coding RNAs (lncRNAs), and exosomal RNAs, to modulate pathogenic molecular pathways and address the underlying molecular origins of neurodegeneration. We evaluate the integration of advanced computational techniques for RNA structure prediction and gene regulatory network analysis, alongside chemical engineering strategies-such as Locked Nucleic Acids (LNAs) and phosphorothioate modifications-aimed at enhancing the stability and specificity of RNA-based molecules. Furthermore, we analyze cutting-edge delivery and editing technologies, including nanotechnology-driven solutions for precise neuronal targeting and the CRISPR/Cas13 system for direct ncRNA manipulation.The findings indicate that while challenges in delivery efficiency and long-term efficacy persist, the synergy of chemical engineering and computational modeling significantly improves the therapeutic profile of ncRNAs, with exosomal pathways offering a novel route for intercellular signaling modulation and biomarker discovery. Therapeutic interventions directed at specific clinical targets, such as miR-34a and BACE1-AS, demonstrate the capacity to influence protein aggregation and neuroinflammatory cascades. Although ncRNA-based therapies are currently in nascent stages, ongoing technological advancements in RNA editing and nanotechnology offer a transformative framework that could redefine the future of ND treatment and successfully halt disease progression rather than merely managing symptoms.
    • Contributed Indexing:
      Keywords: CRISPR/Cas13; RNA therapeutics; blood–brain barrier; exosomes; gene regulation; nanotechnology; ncRNA; neurodegenerative diseases; non-coding RNAs; precision medicine
    • Accession Number:
      0 (RNA, Untranslated)
      0 (MicroRNAs)
      0 (RNA, Long Noncoding)
    • Publication Date:
      Date Created: 20260414 Date Completed: 20260414 Latest Revision: 20260416
    • Publication Date:
      20260416
    • Accession Number:
      PMC13073327
    • Accession Number:
      10.3390/ijms27073260
    • Accession Number:
      41977439