Autophagy in motor neuron disease: Key pathogenetic mechanisms and therapeutic targets

Maria Sara Cipolat Mis, Simona Brajkovic, Emanuele Frattini, Alessio Di Fonzo, Stefania Corti

Research output: Contribution to journalArticlepeer-review


Autophagy is a lysosome-dependant intracellular degradation process that eliminates long-lived proteins as well as damaged organelles from the cytoplasm. An increasing body of evidence suggests that dysregulation of this system plays a pivotal role in the etiology and/or progression of neurodegenerative diseases including motor neuron disorders. Herein, we review the latest findings that highlight the involvement of autophagy in the pathogenesis of amyotrophic lateral sclerosis (ALS) and the potential role of this pathway as a target of therapeutic purposes. Autophagy promotes the removal of toxic, cytoplasmic aggregate-prone pathogenetic proteins, enhances cell survival, and modulates inflammation. The existence of several drugs targeting this pathway can facilitate the translation of basic research to clinical trials for ALS and other motor neuron diseases.

Original languageEnglish
Pages (from-to)84-90
Number of pages7
JournalMolecular and Cellular Neuroscience
Publication statusPublished - Apr 1 2016


  • Amyotrophic lateral sclerosis
  • Authophagy
  • Protein aggregation

ASJC Scopus subject areas

  • Molecular Biology
  • Cellular and Molecular Neuroscience
  • Cell Biology


Dive into the research topics of 'Autophagy in motor neuron disease: Key pathogenetic mechanisms and therapeutic targets'. Together they form a unique fingerprint.

Cite this