Login / Signup

Gene-based therapies for neuromuscular disorders.

Edmar ZanoteliMarcondes Cavalcante de França JuniorOswaldo Wiliam Marques Junior
Published in: Arquivos de neuro-psiquiatria (2024)
Neuromuscular diseases (NMD) include a broad group of medical conditions with both acquired and genetic causes. In recent years, important advances have been made in the treatment of genetically caused NMD, and most of these advances are due to the implementation of therapies aimed at gene regulation. Among these therapies, gene replacement, small interfering RNA (siRNA), and antisense antinucleotides are the most promising approaches. More importantly, some of these therapies have already gained regulatory approval or are in the final stages of approval. The review focuses on motor neuron diseases, neuropathies, and Duchenne muscular dystrophy, summarizing the most recent developments in gene-based therapies for these conditions.
Keyphrases
  • duchenne muscular dystrophy
  • genome wide
  • copy number
  • healthcare
  • primary care
  • genome wide identification
  • transcription factor
  • dna methylation
  • drug delivery
  • nucleic acid
  • muscular dystrophy