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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.
Abstract:
This review will briefly introduce microRNAs (miRNAs) and dissect their contribution to multiple sclerosis (MS) and its clinical outcomes. For this purpose, we provide a concise overview of the present knowledge of MS pathophysiology, biomarkers and treatment options, delving into the role of selectively expressed miRNAs in clinical forms of this disease, as measured in several biofluids such as serum, plasma or cerebrospinal fluid (CSF). Additionally, up-to-date information on current strategies applied to miRNA-based therapeutics will be provided, including miRNA restoration therapy (lentivirus expressing a specific type of miRNA and miRNA mimic) and miRNA inhibition therapy such as antisense oligonucleotides, small molecules inhibitors, locked nucleic acids (LNAs), anti-miRNAs, and antagomirs. Finally, it will highlight future directions and potential limitations associated with their application in MS therapy, emphasizing the need for improved delivery methods and validation of therapeutic efficacy.
Insights
MicroRNAs (miRNAs) are key players in multiple sclerosis (MS) pathogenesis and outcomes. This review explores their diagnostic potential and therapeutic applications for MS.
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.
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