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Neurodegenerative Disorders Treatment: The MicroRNA Role.
Curr Gene Ther. 2017; 17(5):327-363.CG

Abstract

Neurodegenerative diseases such as Alzheimer's disease, Parkinson's disease, amyotrophic lateral sclerosis, Huntington's disease and prion disease are not timely and effectively treated using conventional therapies. This emphasizes the need for alternative therapeutic approaches. In this respect, gene-based therapies have been adopted as potentially feasible alternative therapies, where the microRNA (miRNA) approach has experienced a great explosion in recent years. Because miRNAs have been shown to be implicated in the pathogenesis of several diseases including neurodegenerative diseases, they are intensely studied as candidates for diagnostic and prognostic biomarkers, as predictors of drug response and as therapeutic agents. In this review, we evaluate the feasibility of both direct and indirect miRNA mimics and inhibitors toward the regulation of neurodegenerative-related genes both in vivo and in vitro models, highlight the advantages and drawbacks associated with miRNA-based therapy, and summarize the relevant techniques and approaches attempted to deliver miRNAs to the central nervous system for therapeutic purposes, with particular regard to the exosomes. Additionally, we describe a new approach that holds great promise for the treatment of a wide range of diseases including neurodegenerative disorders. This approach is based on addressing the incorporation of miRNAs into exosomes to increase the quantity and quality of miRNA packed and delivered to the central nervous system and other sites of action.

Authors+Show Affiliations

National Center for Global Health, Istituto Superiore di Sanita, Rome, Italy.National Center for Drug Research and Evaluation, Istituto Superiore di Sanita, Rome, Italy.

Pub Type(s)

Journal Article
Review

Language

eng

PubMed ID

29357791

Citation

Ridolfi, Barbara, and Hanin Abdel-Haq. "Neurodegenerative Disorders Treatment: the MicroRNA Role." Current Gene Therapy, vol. 17, no. 5, 2017, pp. 327-363.
Ridolfi B, Abdel-Haq H. Neurodegenerative Disorders Treatment: The MicroRNA Role. Curr Gene Ther. 2017;17(5):327-363.
Ridolfi, B., & Abdel-Haq, H. (2017). Neurodegenerative Disorders Treatment: The MicroRNA Role. Current Gene Therapy, 17(5), 327-363. https://doi.org/10.2174/1566523218666180119120726
Ridolfi B, Abdel-Haq H. Neurodegenerative Disorders Treatment: the MicroRNA Role. Curr Gene Ther. 2017;17(5):327-363. PubMed PMID: 29357791.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Neurodegenerative Disorders Treatment: The MicroRNA Role. AU - Ridolfi,Barbara, AU - Abdel-Haq,Hanin, PY - 2017/09/04/received PY - 2017/12/29/revised PY - 2018/01/11/accepted PY - 2018/1/24/pubmed PY - 2019/4/2/medline PY - 2018/1/24/entrez KW - Exosomes KW - MicroRNA KW - Neurodegenerative diseases KW - Therapy. KW - miRNA delivery KW - miRNA inhibitors KW - miRNA mimics KW - miRNA-based therapy SP - 327 EP - 363 JF - Current gene therapy JO - Curr Gene Ther VL - 17 IS - 5 N2 - Neurodegenerative diseases such as Alzheimer's disease, Parkinson's disease, amyotrophic lateral sclerosis, Huntington's disease and prion disease are not timely and effectively treated using conventional therapies. This emphasizes the need for alternative therapeutic approaches. In this respect, gene-based therapies have been adopted as potentially feasible alternative therapies, where the microRNA (miRNA) approach has experienced a great explosion in recent years. Because miRNAs have been shown to be implicated in the pathogenesis of several diseases including neurodegenerative diseases, they are intensely studied as candidates for diagnostic and prognostic biomarkers, as predictors of drug response and as therapeutic agents. In this review, we evaluate the feasibility of both direct and indirect miRNA mimics and inhibitors toward the regulation of neurodegenerative-related genes both in vivo and in vitro models, highlight the advantages and drawbacks associated with miRNA-based therapy, and summarize the relevant techniques and approaches attempted to deliver miRNAs to the central nervous system for therapeutic purposes, with particular regard to the exosomes. Additionally, we describe a new approach that holds great promise for the treatment of a wide range of diseases including neurodegenerative disorders. This approach is based on addressing the incorporation of miRNAs into exosomes to increase the quantity and quality of miRNA packed and delivered to the central nervous system and other sites of action. SN - 1875-5631 UR - https://www.unboundmedicine.com/medline/citation/29357791/Neurodegenerative_Disorders_Treatment:_The_MicroRNA_Role_ DB - PRIME DP - Unbound Medicine ER -