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

ABSTRACT: 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

SUBMITTER: Andreana I 

PROVIDER: S-EPMC7922331 | biostudies-literature | 2021 Feb

REPOSITORIES: biostudies-literature