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Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents
Published on: August 10, 2018
Emerging role of microRNAs in the pathogenesis of amyotrophic lateral sclerosis
Nader Akbari Dilmaghani1, Bashdar Mahmud Hussen2, Saeedeh Nateghinia1
1Skull Base Research Center, Loghman Hakim Hospital, Shahid Beheshti University of Medical Sciences, Tehran, Iran.
Abstract:
Amyotrophic lateral sclerosis (ALS) is a deadly motor neuron disease (MND) and the most frequent MND in adults. ALS is recognized by degenerative alterations in both upper and lower motor neurons. This disorder is classified to familial and sporadic classes. Disease-causing mutations in SOD1, C9ORF72, FUS, and TARDBP have been recognized in familial ALS cases. However, in spite of conduction of several genetic association studies, heritable genetic risk elements in sporadic have not been identified completely. Several miRNAs have been dysregulated in the serum samples or brain tissues of ALS patients. Moreover, a number of miRNAs have been suggested as putative biomarkers for sporadic ALS. In the current manuscript, we review of miRNAs in the development of ALS.
Insights
Amyotrophic lateral sclerosis (ALS), a motor neuron disease, involves motor neuron degeneration. MicroRNAs (miRNAs) show dysregulation in ALS patients and may serve as biomarkers for sporadic forms of the disease.
Area of Science:
- Neuroscience
- Genetics
- Molecular Biology
Background:
- Amyotrophic lateral sclerosis (ALS) is the most common adult motor neuron disease (MND), characterized by upper and lower motor neuron degeneration.
- ALS is categorized into familial and sporadic forms, with known genetic mutations (SOD1, C9ORF72, FUS, TARDBP) identified in familial cases.
- Heritable genetic risk factors for sporadic ALS remain incompletely understood despite extensive research.
Purpose of the Study:
- To review the role of microRNAs (miRNAs) in the pathogenesis of Amyotrophic Lateral Sclerosis (ALS).
- To explore the potential of miRNAs as biomarkers for sporadic ALS.
- To synthesize current understanding of miRNA dysregulation in ALS development.
Main Methods:
- Literature review of studies investigating microRNAs in Amyotrophic Lateral Sclerosis.
- Analysis of research on genetic associations and biomarkers in ALS.
- Synthesis of findings related to miRNA expression in ALS patient samples and tissues.
Main Results:
- Several microRNAs (miRNAs) have been observed to be dysregulated in both serum samples and brain tissues of ALS patients.
- Specific miRNAs have been proposed as potential diagnostic or prognostic biomarkers for sporadic ALS.
- The review highlights the emerging significance of miRNAs in understanding ALS pathophysiology.
Conclusions:
- MicroRNAs play a significant role in the development and progression of Amyotrophic Lateral Sclerosis.
- Dysregulated miRNAs present a promising avenue for the identification of biomarkers for sporadic ALS.
- Further research into miRNA mechanisms is crucial for advancing ALS diagnostics and therapeutics.
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