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Updated: Nov 7, 2025

Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents
Published on: August 10, 2018
MiRNAs and Muscle Regeneration: Therapeutic Targets in Duchenne Muscular Dystrophy
Amelia Eva Aránega1,2, Estefanía Lozano-Velasco1,2, Lara Rodriguez-Outeiriño1,2
1Department of Experimental Biology, Faculty of Experimental Sciences, University of Jaen, Paraje Las Lagunillas s/n, 23009 Jaen, Spain.
Abstract:
microRNAs (miRNAs) are small non-coding RNAs required for the post-transcriptional control of gene expression. MicroRNAs play a critical role in modulating muscle regeneration and stem cell behavior. Muscle regeneration is affected in muscular dystrophies, and a critical point for the development of effective strategies for treating muscle disorders is optimizing approaches to target muscle stem cells in order to increase the ability to regenerate lost tissue. Within this framework, miRNAs are emerging as implicated in muscle stem cell response in neuromuscular disorders and new methodologies to regulate the expression of key microRNAs are coming up. In this review, we summarize recent advances highlighting the potential of miRNAs to be used in conjunction with gene replacement therapies, in order to improve muscle regeneration in the context of Duchenne Muscular Dystrophy (DMD).
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