Autophagy and Neurodegeneration: Pathogenic Mechanisms and Therapeutic Opportunities

Fiona M. Menzies, Angeleen Fleming, Andrea Caricasole, Carla F. Bento, Stephen P. Andrews, Avraham Ashkenazi, Jens Füllgrabe, Anne Jackson, Maria Jimenez Sanchez, Cansu Karabiyik, Floriana Licitra, Ana Lopez Ramirez, Mariana Pavel, Claudia Puri, Maurizio Renna, Thomas Ricketts, Lars Schlotawa, Mariella Vicinanza, Hyeran Won, Ye ZhuJohn Skidmore, David C. Rubinsztein*

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review


Autophagy is a conserved pathway that delivers cytoplasmic contents to the lysosome for degradation. Here we consider its roles in neuronal health and disease. We review evidence from mouse knockout studies demonstrating the normal functions of autophagy as a protective factor against neurodegeneration associated with intracytoplasmic aggregate-prone protein accumulation as well as other roles, including in neuronal stem cell differentiation. We then describe how autophagy may be affected in a range of neurodegenerative diseases. Finally, we describe how autophagy upregulation may be a therapeutic strategy in a wide range of neurodegenerative conditions and consider possible pathways and druggable targets that may be suitable for this objective.

Original languageEnglish
Pages (from-to)1015-1034
Number of pages20
Issue number5
StatePublished - 8 Mar 2017
Externally publishedYes


  • Alzheimer's disease
  • Huntington's disease
  • Parkinson's disease
  • autophagy
  • dementia
  • lysosome
  • motor neuron disease
  • neurodegeneration
  • tau


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