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Published on: August 10, 2018
Opportunities Offered by Graphene Nanoparticles for MicroRNAs Delivery for Amyotrophic Lateral Sclerosis Treatment
Benedetta Niccolini1, Valentina Palmieri1,2,3, Marco De Spirito1,2
1Dipartimento di Neuroscienze, Università Cattolica del Sacro Cuore, 00168 Rome, Italy.
Abstract:
Amyotrophic lateral sclerosis (ALS) is characterized by the degeneration and death of motor neurons. This neurodegenerative disease leads to muscle atrophy, paralysis, and death due to respiratory failure. MicroRNAs (miRNAs) are small non-coding ribonucleic acids (RNAs) with a length of 19 to 25 nucleotides, participating in the regulation of gene expression. Different studies have demonstrated that miRNAs deregulation is critical for the onset of a considerable number of neurodegenerative diseases, including ALS. Some studies have underlined how miRNAs are deregulated in ALS patients and for this reason, design therapies are used to correct the aberrant expression of miRNAs. With this rationale, delivery systems can be designed to target specific miRNAs. Specifically, these systems can be derived from viral vectors (viral systems) or synthetic or natural materials, including exosomes, lipids, and polymers. Between many materials used for non-viral vectors production, the two-dimensional graphene and its derivatives represent a good alternative for efficiently delivering nucleic acids. The large surface-to-volume ratio and ability to penetrate cell membranes are among the advantages of graphene. This review focuses on the specific pathogenesis of miRNAs in ALS and on graphene delivery systems designed for gene delivery to create a primer for future studies in the field.
Insights
MicroRNAs (miRNAs) are implicated in Amyotrophic Lateral Sclerosis (ALS) pathogenesis. Graphene-based delivery systems offer a promising strategy for correcting aberrant miRNA expression in ALS treatment.
Area of Science:
- Neuroscience
- Molecular Biology
- Biotechnology
Background:
- Amyotrophic Lateral Sclerosis (ALS) involves motor neuron degeneration, leading to paralysis and respiratory failure.
- MicroRNA (miRNA) deregulation is a key factor in the development of neurodegenerative diseases like ALS.
- Current therapeutic strategies aim to correct aberrant miRNA expression in ALS patients.
Purpose of the Study:
- To review the role of miRNA dysregulation in ALS pathogenesis.
- To explore the potential of graphene-based delivery systems for miRNA-targeted gene therapy in ALS.
- To provide a foundation for future research in this field.
Main Methods:
- Literature review focusing on miRNA involvement in ALS.
- Analysis of graphene and its derivatives as non-viral vectors for nucleic acid delivery.
- Discussion of the advantages of graphene for gene delivery applications.
Main Results:
- miRNA deregulation is a significant contributor to ALS pathology.
- Graphene's properties, such as high surface-to-volume ratio and cell membrane penetration, make it suitable for gene delivery.
- Graphene-based systems present a viable non-viral alternative for delivering nucleic acids.
Conclusions:
- Targeting miRNA dysregulation holds therapeutic potential for ALS.
- Graphene delivery systems offer an efficient and promising approach for miRNA-based gene therapy in ALS.
- Further research into graphene-mediated miRNA delivery is warranted for ALS treatment development.
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