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Targeting Non-Coding RNAs as a Potential Therapeutic and Delivery Strategy Against Neurodegenerative Diseases.
1Medical School, National and Kapodistrian University of Athens, M. Asias 75, 11574 Athens, Greece.
International Journal of Molecular Sciences
|April 14, 2026
Summary
Non-coding RNAs (ncRNAs) show promise for treating neurodegenerative diseases (NDs) by targeting molecular pathways. Advanced computational and chemical engineering strategies enhance ncRNA therapies, potentially halting disease progression.
Area of Science:
- Biochemistry
- Genetics
- Neuroscience
Background:
- Neurodegenerative diseases (NDs) pose a significant global health challenge due to progressive neuronal loss and limited disease-modifying treatments.
- Current therapeutic strategies primarily focus on symptom management rather than addressing the root molecular causes of NDs.
Purpose of the Study:
- To review the therapeutic potential of non-coding RNAs (ncRNAs) in neurodegenerative diseases.
- To explore the integration of computational techniques and chemical engineering for ncRNA-based therapies.
- To analyze advanced delivery and editing technologies for ncRNA manipulation in the nervous system.
Main Methods:
- Literature review of ncRNA therapeutics for NDs.
- Evaluation of computational methods for RNA structure and gene networks.
- Analysis of chemical engineering strategies (e.g., LNAs) for RNA stability.
- Assessment of nanotechnology and CRISPR/Cas13 for ncRNA delivery and editing.
Main Results:
- ncRNAs, including miRNAs, lncRNAs, and exosomal RNAs, can modulate pathogenic pathways in NDs.
- Computational modeling and chemical engineering enhance the stability and specificity of ncRNA therapeutics.
- Exosomal pathways offer potential for intercellular signaling modulation and biomarker discovery.
- Specific ncRNAs like miR-34a and BACE1-AS show potential in influencing protein aggregation and neuroinflammation.
Conclusions:
- ncRNA-based therapies represent a promising frontier for treating neurodegenerative diseases.
- Synergistic approaches combining computational, chemical, and nanotechnological strategies are crucial for therapeutic advancement.
- Despite challenges in delivery and efficacy, ncRNA therapies hold the potential to halt ND progression, moving beyond symptomatic treatment.
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