Research advances in gene therapy approaches for the treatment of amyotrophic lateral sclerosis

Monica Nizzardo, Chiara Simone, Marianna Falcone, Giulietta Riboldi, Federica Rizzo, Francesca Magri, Nereo Bresolin, Giacomo P. Comi, Stefania Corti

Research output: Contribution to journalArticlepeer-review


Amyotrophic lateral sclerosis (ALS) is a devastating neurodegenerative disease of motor neurons that causes progressive muscle weakness, paralysis, and premature death. No effective therapy is available. Research in the motor neuron field continues to grow, and recent breakthroughs have demonstrated the possibility of completely achieving rescue in animal models of spinal muscular atrophy, a genetic motor neuron disease. With adeno-associated virus (AAV) vectors, gene transfer can be achieved with systemic non-invasive injection and minimal toxicity. In the context of this success, we review gene therapy approaches for ALS, considering what has been done and the possible future directions for effective application of the latest generation of vectors for clinical translation. We focus on recent developments in the areas of RNA/antisense-mediated silencing of specific ALS causative genes like superoxide dismutase-1 and other molecular pathogenetic targets, as well as the administration of neuroprotective factors with viral vectors. We argue that gene therapy offers new opportunities to open the path for clinical progress in treating ALS.

Original languageEnglish
Pages (from-to)1641-1650
Number of pages10
JournalCellular and Molecular Life Sciences
Issue number10
Publication statusPublished - May 2012


  • Adeno-associated vectors
  • Amyotrophic lateral sclerosis
  • Gene therapy
  • RNA interference

ASJC Scopus subject areas

  • Cell Biology
  • Molecular Biology
  • Molecular Medicine
  • Pharmacology
  • Cellular and Molecular Neuroscience


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