Advances in treating amyotrophic lateral sclerosis: Insights from pathophysiological studies

Steve Vucic, Jeffrey D. Rothstein, Matthew C. Kiernan

Research output: Contribution to journalReview articlepeer-review


Amyotrophic lateral sclerosis (ALS) is the most frequently occurring of the neuromuscular degenerative disorders, with a median survival time of 3-5 years. The pathophysiological mechanisms underlying ALS are multifactorial, with a complex interaction between genetic factors and molecular pathways. To date 16 genes and loci have been associated with ALS, with mutations in DNA/RNA-regulating genes including the recently described c9orf72 (chromosome 9 open reading frame 72) gene, suggesting an important role for dysregulation of RNA metabolism in ALS pathogenesis. Further, dysfunction of molecular pathways, including glutamate-mediated excitotoxicity, has been identified in sporadic and familial ALS, indicating the existence of a common pathogenic pathway. These pathophysiological insights have suggested novel therapeutic approaches, including stem cell and genetics-based strategies, providing hope for feasible treatment of ALS.

Original languageEnglish (US)
Pages (from-to)433-442
Number of pages10
JournalTrends in neurosciences
Issue number8
StatePublished - Aug 2014

ASJC Scopus subject areas

  • Neuroscience(all)


Dive into the research topics of 'Advances in treating amyotrophic lateral sclerosis: Insights from pathophysiological studies'. Together they form a unique fingerprint.

Cite this