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Updated: Jun 13, 2025

Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents

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Published on: August 10, 2018

MiRNA-based therapeutic potential in multiple sclerosis.

Ana Zabalza¹, Agustin Pappolla¹, Manuel Comabella¹

¹Vall Hebron University Hospital & Research Institute (VHIR), Multiple Sclerosis Centre of Catalonia (Cemcat) & Neurology Department, Universitat Autonoma de Barcelona, Barcelona, Spain.

Frontiers in Immunology

|September 13, 2024

Summary

This summary is machine-generated.

MicroRNAs (miRNAs) are key players in multiple sclerosis (MS) pathogenesis and outcomes. This review explores their diagnostic potential and therapeutic applications for MS.

Keywords:

biomarkers microRNAs multiple sclerosis neurology therapeutic targets

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Area of Science:

Neuroscience
Molecular Biology
Genetics

Background:

Multiple sclerosis (MS) is a chronic autoimmune disease affecting the central nervous system.
Current understanding of MS pathophysiology, biomarkers, and treatments is evolving.
MicroRNAs (miRNAs) are small non-coding RNAs with significant regulatory roles in gene expression.

Purpose of the Study:

To review the role of microRNAs (miRNAs) in multiple sclerosis (MS) and its clinical outcomes.
To explore the potential of miRNAs as biomarkers for MS diagnosis and prognosis.
To discuss current and emerging miRNA-based therapeutic strategies for MS.

Main Methods:

Literature review of studies investigating miRNAs in MS.
Analysis of miRNA expression in biofluids (serum, plasma, cerebrospinal fluid).
Overview of miRNA-based therapeutic approaches, including restoration and inhibition.

Main Results:

Specific miRNAs are differentially expressed in various clinical forms of MS.
miRNAs show potential as diagnostic and prognostic biomarkers in MS.
Various miRNA-based therapeutic strategies are under development for MS.

Conclusions:

miRNAs represent promising therapeutic targets and biomarkers for multiple sclerosis (MS).
Further research is needed to validate therapeutic efficacy and optimize delivery methods for miRNA-based treatments.
Addressing limitations in delivery and validation is crucial for advancing miRNA therapeutics in MS.