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MicroRNAs (miRs) in Muscle Gene Therapy

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Muscle Gene Therapy

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Abstract

Despite recent advances in scientific knowledge and several clinical trials, muscle gene and cell therapies remain a major challenge. As a matter of fact, novel technologies are being developed for targeting muscle tissues including CRISPR, TALEN, and iPS technologies indicating that gene-based therapies still hold significant promises. Recent findings from our laboratory and others unveiled that microRNAs (miRs), small nonprotein-coding RNAs, are able to posttranscriptionally regulate many genes and exert pleiotropic effects in the muscle. Deleterious changes in miR expression play an important role in muscle diseases. In this regard, miRs are possible therapeutic targets, and miR-based gene therapy for smooth, skeletal, and cardiac muscles is an extremely interesting field for harnessing the complexity of miR-based therapeutic approaches. In this chapter, we will focus on miR-driven regulation of myogenic routes in homeostatic and challenging states. We will also survey the intriguing perspective of miR biological transfers, including the delivery of functional miRs via exosomes that unlike other vectors are cell-free natural systems for ferrying RNAs between cells. Finally, we will review the recent literature on key miR targets to treat skeletal, cardiac, and smooth muscle diseases and novel valuable clinical tools for more effective treatment strategies in muscle degeneration.

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Authors and Affiliations

  1. Translational Cardiomyology, Stem Cell Research Institute, Stem Cell Biology and Embryology Unit, Department of Development and Regeneration, KU Leuven, Leuven, Belgium

    Alessio Rotini, Giorgia Giacomazzi, Ester Sara Di Filippo & Maurilio Sampaolesi

  2. Interuniversity Institute of Myology, Chieti, Italy

    Alessio Rotini, Giorgia Giacomazzi, Ester Sara Di Filippo & Maurilio Sampaolesi

  3. Department of Neuroscience, Imaging and Clinical Sciences, University “G. d’Annunzio” Chieti-Pescara, Chieti, Italy

    Alessio Rotini & Ester Sara Di Filippo

  4. Human Anatomy Unit, Department of Public Health, Experimental and Forensic Medicine, University of Pavia, Pavia, Italy

    Maurilio Sampaolesi

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  1. Alessio Rotini
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  2. Giorgia Giacomazzi
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  3. Ester Sara Di Filippo
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  4. Maurilio Sampaolesi
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Correspondence to Maurilio Sampaolesi .

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Editors and Affiliations

  1. Department of Molecular Microbiology and Immunology, School of Medicine, University of Missouri, Columbia, MO, USA; Department of Neurology, School of Medicine, University of Missouri, Columbia, MO, USA, Department of Biomedical Sciences College of Veterinary Medicine, University of Missouri, Columbia, MO, USA; Department of Bioengineering, University of Missouri, Columbia, MO, USA

    Dongsheng Duan

  2. Department of Pediatrics Nationwide Children’s Hospital and Research Institute, Department of Neurology Nationwide Children’s Hospital and Research Institute, The Ohio State University, Columbus, OH, USA

    Jerry R. Mendell

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Rotini, A., Giacomazzi, G., Di Filippo, E.S., Sampaolesi, M. (2019). MicroRNAs (miRs) in Muscle Gene Therapy. In: Duan, D., Mendell, J. (eds) Muscle Gene Therapy. Springer, Cham. https://doi.org/10.1007/978-3-030-03095-7_6

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