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Teaching an Old Molecule New Tricks: Drug Repositioning for Duchenne Muscular Dystrophy

1
Department of Biology, University of Padova, via U. Bassi 58/B, 35131 Padova, Italy
2
Interuniversity Institute of Myology (IIM), Administrative headquarters University of Perugia, Piazza Lucio Severi 1, 06132, Perugia, Italy
3
Department of Biomedical Sciences, University of Padova, via U. Bassi 58/B, 35131 Padova, Italy
4
Department of Neurosciences, University of Padova, Via Giustiniani, 5-35128 Padova, Italy
5
Fondazione Istituto di Ricerca Pediatrica Città della Speranza-IRP, Corso Stati Uniti, 4, 35127 Padova, Italy
*
Authors to whom correspondence should be addressed.
Int. J. Mol. Sci. 2019, 20(23), 6053; https://doi.org/10.3390/ijms20236053
Received: 31 October 2019 / Revised: 28 November 2019 / Accepted: 28 November 2019 / Published: 30 November 2019
(This article belongs to the Special Issue Rare Diseases: Molecular Mechanisms and Therapeutic Strategies (II))
Duchenne muscular dystrophy (DMD) is one of the most severe forms of inherited muscular dystrophies. The disease is caused by the lack of dystrophin, a structurally essential protein; hence, a definitive cure would necessarily have to pass through some form of gene and/or cell therapy. Cell- and genetic-based therapeutics for DMD have been explored since the 1990s; recently, two of the latter have been approved for clinical use, but their efficacy is still very low. In parallel, there have been great ongoing efforts aimed at targeting the downstream pathogenic effects of dystrophin deficiency using classical pharmacological approaches, with synthetic or biological molecules. However, as it is always the case with rare diseases, R&D costs for new drugs can represent a major hurdle for researchers and patients alike. This problem can be greatly alleviated by experimenting the use of molecules that had originally been developed for different conditions, a process known as drug repurposing or drug repositioning. In this review, we will describe the state of the art of such an approach for DMD, both in the context of clinical trials and pre-clinical studies. View Full-Text
Keywords: Duchenne muscular dystrophy; drug repurposing; druggable targets; clinical use; mdx mice Duchenne muscular dystrophy; drug repurposing; druggable targets; clinical use; mdx mice
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Vitiello, L.; Tibaudo, L.; Pegoraro, E.; Bello, L.; Canton, M. Teaching an Old Molecule New Tricks: Drug Repositioning for Duchenne Muscular Dystrophy. Int. J. Mol. Sci. 2019, 20, 6053.

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