Key factors highlighting the significance of miRNA-based treatments in neurological disorders
| Significance of miRNA | Clinical outcomes | Conclusion | References |
|---|---|---|---|
| Accurate targeting | miR-379-410 cluster regulates neurogenesis by targeting multiple binding sites in the N-cadherin 3′-UTR. | This additive effect underscores how miRNAs can cooperate to exert stronger repressive actions on their targets, which is crucial during brain development. miRNAs can simultaneously target multiple mRNAs, offering a multi-targeting approach for cancer therapies. | [12] |
| Modulation of complex pathways | Reduced β-amyloid deposition, improved cognitive function, and modulated neuroinflammation using miR-124-3p. | miRNAs can modify multiple molecular pathways, providing a more comprehensive and effective intervention in AD. | [13, 14] |
| Illness modification | Downregulation of CELF2 improved motor function, reduced neurodegeneration, and sustained benefits over time. | miRNA-based treatments can alter the underlying disease process, providing long-term benefits. | [15] |
| Blood-brain barrier (BBB) penetrance | Reduced infarct size by 40%, enhanced neuroprotective signaling, and improved motor function after BBB penetration. | miRNAs can cross the BBB, providing effective therapeutics for central nervous system (CNS) conditions, such as ischemic stroke. | [16] |
| Biomarker possibilities | Downregulation of miR-125b-5p and miR-26b-5p was correlated with the severity of cognitive impairment. | miRNAs can serve as biomarkers for early diagnosis and tracking disease progression in neurological conditions. | [17] |
| Lessened off-target effects | Significant apoptosis in GBM cells with minimal neurotoxicity by targeting the Notch pathway with miR-34a mimics. | miRNA-based therapies offer high specificity, reducing off-target effects and improving safety compared to traditional therapies. | [18] |
| Versatility in delivery | Adeno-associated virus-9 (AAV9)-delivered miR-132 was stably expressed in HD brain regions, silencing MeCP2 and enhancing synaptic plasticity and neuronal survival. | AAV vectors are effective for targeted delivery, and different platforms can be used for diverse neurological disorders. | [19] |
| Personalized medicine | Reduced amyloid plaque deposition and improved cognitive function with personalized miR-29 mimic therapy. | Personalized miRNA therapies can be tailored to individual patients, enhancing treatment efficacy. | [20] |
| Combination therapies | Enhanced efficacy of miR-107, which regulates BACE-1 expression involved in amyloid-beta production. | miRNAs combined with existing therapies can reduce amyloid plaque formation and improve cognitive function in animal models. | [21] |
| Preclinical success | Reduced neuroinflammation, improved motor and cognitive function, and enhanced recovery post-injury using miR-124. | miRNA-based therapies, like miR-124, show promising preclinical results for treating neurological injuries. | [22] |
miRNA-based treatments offer a game-changing strategy to address the intricate and difficult landscape of neurological illnesses. miRNA: microRNA; mRNAs: messenger RNAs; AD: Alzheimer’s disease; HD: Huntington’s disease; BACE-1: beta-site amyloid precursor protein cleaving enzyme 1
SP: Conceptualization, Supervision, Validation, Writing—original draft. SM: Visualization, Writing—review & editing.
The authors declare that they have no conflicts of interest.
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