Nanomedicine for Gene Delivery and Drug Repurposing in the Treatment of Muscular Dystrophies
Muscular Dystrophies (MDs) are a group of rare inherited genetic muscular pathologies encompassing a variety of clinical phenotypes, gene mutations and mechanisms of disease. MDs undergo progressive skeletal muscle degeneration causing severe health problems that lead to poor life quality, disabilit...
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| Format: | Article |
| Language: | English |
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MDPI AG
2021-02-01
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| Series: | Pharmaceutics |
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| Online Access: | https://www.mdpi.com/1999-4923/13/2/278 |
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| author | Ilaria Andreana Mathieu Repellin Flavia Carton David Kryza Stéphanie Briançon Bénédicte Chazaud Rémi Mounier Silvia Arpicco Manuela Malatesta Barbara Stella Giovanna Lollo |
| author_facet | Ilaria Andreana Mathieu Repellin Flavia Carton David Kryza Stéphanie Briançon Bénédicte Chazaud Rémi Mounier Silvia Arpicco Manuela Malatesta Barbara Stella Giovanna Lollo |
| author_sort | Ilaria Andreana |
| collection | DOAJ |
| description | Muscular Dystrophies (MDs) are a group of rare inherited genetic muscular pathologies encompassing a variety of clinical phenotypes, gene mutations and mechanisms of disease. MDs undergo progressive skeletal muscle degeneration causing severe health problems that lead to poor life quality, disability and premature death. There are no available therapies to counteract the causes of these diseases and conventional treatments are administered only to mitigate symptoms. Recent understanding on the pathogenetic mechanisms allowed the development of novel therapeutic strategies based on gene therapy, genome editing CRISPR/Cas9 and drug repurposing approaches. Despite the therapeutic potential of these treatments, once the actives are administered, their instability, susceptibility to degradation and toxicity limit their applications. In this frame, the design of delivery strategies based on nanomedicines holds great promise for MD treatments. This review focuses on nanomedicine approaches able to encapsulate therapeutic agents such as small chemical molecules and oligonucleotides to target the most common MDs such as Duchenne Muscular Dystrophy and the Myotonic Dystrophies. The challenge related to in vitro and in vivo testing of nanosystems in appropriate animal models is also addressed. Finally, the most promising nanomedicine-based strategies are highlighted and a critical view in future developments of nanomedicine for neuromuscular diseases is provided. |
| first_indexed | 2024-03-09T00:43:37Z |
| format | Article |
| id | doaj.art-494a866674c2496da2fd3a422c4cee05 |
| institution | Directory Open Access Journal |
| issn | 1999-4923 |
| language | English |
| last_indexed | 2024-03-09T00:43:37Z |
| publishDate | 2021-02-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Pharmaceutics |
| spelling | doaj.art-494a866674c2496da2fd3a422c4cee052023-12-11T17:38:17ZengMDPI AGPharmaceutics1999-49232021-02-0113227810.3390/pharmaceutics13020278Nanomedicine for Gene Delivery and Drug Repurposing in the Treatment of Muscular DystrophiesIlaria Andreana0Mathieu Repellin1Flavia Carton2David Kryza3Stéphanie Briançon4Bénédicte Chazaud5Rémi Mounier6Silvia Arpicco7Manuela Malatesta8Barbara Stella9Giovanna Lollo10Laboratoire d’Automatique, de Génie des Procédés et de Génie Pharmaceutique, Université Claude Bernard Lyon 1, CNRS UMR 5007, 43 bd 11 Novembre 1918, 69622 Villeurbanne, FranceLaboratoire d’Automatique, de Génie des Procédés et de Génie Pharmaceutique, Université Claude Bernard Lyon 1, CNRS UMR 5007, 43 bd 11 Novembre 1918, 69622 Villeurbanne, FranceDepartment of Neurosciences, Biomedicine and Movement Sciences, Anatomy and Histology Section, University of Verona, Strada Le Grazie 8, 37134 Verona, ItalyLaboratoire d’Automatique, de Génie des Procédés et de Génie Pharmaceutique, Université Claude Bernard Lyon 1, CNRS UMR 5007, 43 bd 11 Novembre 1918, 69622 Villeurbanne, FranceLaboratoire d’Automatique, de Génie des Procédés et de Génie Pharmaceutique, Université Claude Bernard Lyon 1, CNRS UMR 5007, 43 bd 11 Novembre 1918, 69622 Villeurbanne, FranceInstitut NeuroMyoGène, University of Lyon, INSERM U1217, CNRS UMR 5310, 8 Avenue Rockefeller, 69008 Lyon, FranceInstitut NeuroMyoGène, University of Lyon, INSERM U1217, CNRS UMR 5310, 8 Avenue Rockefeller, 69008 Lyon, FranceDepartment of Drug Science and Technology, University of Turin, Via P. Giuria 9, 10125 Torino, ItalyDepartment of Neurosciences, Biomedicine and Movement Sciences, Anatomy and Histology Section, University of Verona, Strada Le Grazie 8, 37134 Verona, ItalyDepartment of Drug Science and Technology, University of Turin, Via P. Giuria 9, 10125 Torino, ItalyLaboratoire d’Automatique, de Génie des Procédés et de Génie Pharmaceutique, Université Claude Bernard Lyon 1, CNRS UMR 5007, 43 bd 11 Novembre 1918, 69622 Villeurbanne, FranceMuscular Dystrophies (MDs) are a group of rare inherited genetic muscular pathologies encompassing a variety of clinical phenotypes, gene mutations and mechanisms of disease. MDs undergo progressive skeletal muscle degeneration causing severe health problems that lead to poor life quality, disability and premature death. There are no available therapies to counteract the causes of these diseases and conventional treatments are administered only to mitigate symptoms. Recent understanding on the pathogenetic mechanisms allowed the development of novel therapeutic strategies based on gene therapy, genome editing CRISPR/Cas9 and drug repurposing approaches. Despite the therapeutic potential of these treatments, once the actives are administered, their instability, susceptibility to degradation and toxicity limit their applications. In this frame, the design of delivery strategies based on nanomedicines holds great promise for MD treatments. This review focuses on nanomedicine approaches able to encapsulate therapeutic agents such as small chemical molecules and oligonucleotides to target the most common MDs such as Duchenne Muscular Dystrophy and the Myotonic Dystrophies. The challenge related to in vitro and in vivo testing of nanosystems in appropriate animal models is also addressed. Finally, the most promising nanomedicine-based strategies are highlighted and a critical view in future developments of nanomedicine for neuromuscular diseases is provided.https://www.mdpi.com/1999-4923/13/2/278nanoparticlesDuchenne Muscular Dystrophymyotonic dystrophyantisense oligonucleotidessmall moleculesCRISPR/Cas9 |
| spellingShingle | Ilaria Andreana Mathieu Repellin Flavia Carton David Kryza Stéphanie Briançon Bénédicte Chazaud Rémi Mounier Silvia Arpicco Manuela Malatesta Barbara Stella Giovanna Lollo Nanomedicine for Gene Delivery and Drug Repurposing in the Treatment of Muscular Dystrophies Pharmaceutics nanoparticles Duchenne Muscular Dystrophy myotonic dystrophy antisense oligonucleotides small molecules CRISPR/Cas9 |
| title | Nanomedicine for Gene Delivery and Drug Repurposing in the Treatment of Muscular Dystrophies |
| title_full | Nanomedicine for Gene Delivery and Drug Repurposing in the Treatment of Muscular Dystrophies |
| title_fullStr | Nanomedicine for Gene Delivery and Drug Repurposing in the Treatment of Muscular Dystrophies |
| title_full_unstemmed | Nanomedicine for Gene Delivery and Drug Repurposing in the Treatment of Muscular Dystrophies |
| title_short | Nanomedicine for Gene Delivery and Drug Repurposing in the Treatment of Muscular Dystrophies |
| title_sort | nanomedicine for gene delivery and drug repurposing in the treatment of muscular dystrophies |
| topic | nanoparticles Duchenne Muscular Dystrophy myotonic dystrophy antisense oligonucleotides small molecules CRISPR/Cas9 |
| url | https://www.mdpi.com/1999-4923/13/2/278 |
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