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Updated: May 3, 2026

Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents
Published on: August 10, 2018
The role of miRNA in motor neuron disease
Min Jeong Kye1, Inês do Carmo G Gonçalves1
1Institute of Human Genetics, University of Cologne Cologne, Germany ; Institute for Genetics, University of Cologne Cologne, Germany.
Abstract:
microRNA is a subset of endogenous non-coding RNA. It binds to partially complementary sequences in mRNAs and inhibits mRNA translation by either blocking translational machinery or degrading mRNAs. It is involved in various cellular processes including cell cycle, development, metabolism, and synaptic plasticity. Dysregulation of miRNA expression and function is reported in various diseases including cancer, metabolic disorders as well as neurological disorders. In nervous system, miRNA related pathways play a very important role in development and function of neuronal cells. Moreover, numerous evidences suggest that dysregulated miRNA related pathways contribute to pathology of neurological disorders such as Alzheimer's disease, amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA). Here, we review current knowledge about the role of miRNAs in motor neuron disorders, especially about two common diseases: SMA and ALS.
Insights
microRNAs regulate gene expression and are vital for nervous system development. Dysregulation of these small RNAs contributes to motor neuron diseases like spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS).
Area of Science:
- Molecular Biology
- Neuroscience
- Genetics
Background:
- microRNAs (miRNAs) are endogenous non-coding RNAs regulating gene expression.
- miRNA dysregulation is implicated in various diseases, including neurological disorders.
- miRNA pathways are crucial for neuronal development and function.
Purpose of the Study:
- To review the role of miRNAs in motor neuron disorders.
- To focus on the involvement of miRNAs in spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS).
Main Methods:
- Literature review of current knowledge on miRNAs in motor neuron disorders.
- Analysis of studies investigating miRNA expression and function in SMA and ALS.
Main Results:
- miRNAs play significant roles in the pathology of SMA and ALS.
- Specific miRNAs and their targets are associated with disease mechanisms.
- Evidence suggests miRNAs as potential therapeutic targets for motor neuron diseases.
Conclusions:
- miRNAs are critical regulators in motor neuron function and development.
- Aberrant miRNA activity contributes to the pathogenesis of SMA and ALS.
- Further research into miRNA pathways may yield novel therapeutic strategies for motor neuron disorders.
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