Advanced Gene-Targeting Therapies for Motor Neuron Diseases and Muscular Dystrophies.

ABSTRACT: Gene therapy is a revolutionary, cutting-edge approach to permanently ameliorate or amend many neuromuscular diseases by targeting their genetic origins. Motor neuron diseases and muscular dystrophies, whose genetic causes are well known, are the frontiers of this research revolution. Several genetic treatments, with diverse mechanisms of action and delivery methods, have been approved during the past decade and have demonstrated remarkable results. However, despite the high number of genetic treatments studied preclinically, those that have been advanced to clinical trials are significantly fewer. The most clinically advanced treatments include adeno-associated virus gene replacement therapy, antisense oligonucleotides, and RNA interference. This review provides a comprehensive overview o

SUBMITTER: Chamakioti M 

PROVIDER: S-EPMC9101723 | biostudies-literature | 2022 Apr

REPOSITORIES: biostudies-literature