Article Dans Une Revue Pharmaceutics Année : 2021

Nanomedicine for Gene Delivery and Drug Repurposing in the Treatment of Muscular Dystrophies

Ilaria Andreana
  • Fonction : Auteur
Flavia Carton
  • Fonction : Auteur
David Kryza
  • Fonction : Auteur
Stéphanie Briançon
Bénédicte Chazaud
Rémi Mounier
Silvia Arpicco
  • Fonction : Auteur
Manuela Malatesta
Barbara Stella
Giovanna Lollo
  • Fonction : Auteur

Résumé

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.

Dates et versions

hal-04749858 , version 1 (23-10-2024)

Identifiants

Citer

Ilaria Andreana, Mathieu Repellin, Flavia Carton, David Kryza, Stéphanie Briançon, et al.. Nanomedicine for Gene Delivery and Drug Repurposing in the Treatment of Muscular Dystrophies. Pharmaceutics, 2021, 13 (2), pp.278. ⟨10.3390/pharmaceutics13020278⟩. ⟨hal-04749858⟩
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