Title:Neurodegenerative Disorders Treatment: The MicroRNA Role
VOLUME: 17 ISSUE: 5
Author(s):Barbara Ridolfi and Hanin Abdel-Haq*
Affiliation:National Center for Global Health, Istituto Superiore di Sanita, Rome, National Center for Drug Research and Evaluation, Istituto Superiore di Sanita, Rome
Keywords:MicroRNA, Neurodegenerative diseases, miRNA-based therapy, Exosomes, miRNA delivery, miRNA mimics,
miRNA inhibitors, Therapy.
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.
