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  4. miR-124: A Promising Therapeutic Target for Central Nervous System Injuries and Diseases

miR-124: A Promising Therapeutic Target for Central Nervous System Injuries and Diseases

Cellular and Molecular Neurobiology, 2022 · DOI: https://doi.org/10.1007/s10571-021-01091-6 · Published: April 22, 2021

PharmacologyNeurologyGenetics

Simple Explanation

Central nervous system injuries and diseases often lead to death or long-term disability. MicroRNAs regulate posttranscriptional gene expression in both physiological and pathological cellular processes, including central nervous system injuries and disorders. Studies on miR-124, one of the most abundant microRNAs in the central nervous system, have shown that its dysregulation is related to the occurrence and development of pathology within the central nervous system. This review provides novel insights into the therapeutic target potential of miR-124 in the treatment of human central nervous system injuries or diseases.

Study Duration
Not specified
Participants
Not specified
Evidence Level
Review Paper

Key Findings

  • 1
    miR-124 acts as one of the key miRNAs regulating neuroinflammation in various CNS pathologies, which may shed light on novel therapies targeting miR-124 for treating CNS insults.
  • 2
    miR-124 has been shown to play an important role in promoting neurogenesis under physiological and pathological conditions.
  • 3
    miR-124 has a comprehensive anti-inflammatory and neuroprotective effect when it acts on groups of cells within the CNS in the process of the neuroinflammatory response; thus, it is promising for the treatment of CNS injuries and diseases.

Research Summary

This review provides an overview of miR-124, its roles in CNS development, and summarizes recent data of its role in neuroinflammation, ischemic stroke, SCI, NDDs, GBM, MS, and the methods for the delivery of miR-124. The mechanisms underlying miR-124 actions are also discussed. Finally, the potential of miR-124 as a therapeutic target to ameliorate CNS injuries and diseases is highlighted. The challenge lies in the productive exploitation of miRNA-based therapeutics, including designing a miR-124 delivery system that can cross the BBB.

Practical Implications

Therapeutic Target for CNS Injuries

miR-124 shows promise as a therapeutic target for central nervous system injuries and diseases due to its regulatory role in neuroinflammation and neurogenesis.

Drug Delivery Systems

Exosomes, nanoparticles, and adeno-associated viruses (AAV) are potential delivery systems for miR-124-based drugs to the CNS, overcoming the blood-brain barrier.

Personalized Medicine

Future research should focus on determining therapeutic windows, modes of treatment, and pharmacokinetic characteristics for individual patients to optimize miR-124-based therapies.

Study Limitations

  • 1
    Disease-specific alterations of miR-124 in the CNS await further investigation.
  • 2
    Designing a miR-124 delivery system that can cross the BBB.
  • 3
    Enabling tissue/cell-specific targeting, while conferring higher stability and avoiding potential off-target effects

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