PERK: A novel therapeutic target for neurodegenerative diseases?

Tao Ma, Eric Klann

Research output: Contribution to journalArticlepeer-review


Identification of therapeutic targets based on novel mechanistic studies is urgently needed for neurodegenerative diseases such as Alzheimer's disease, Parkinson's disease, and prion disease. Multiple lines of evidence have emerged to suggest that inhibition of the stress-induced endoplasmic reticulum kinase PERK (protein kinase RNA-like endoplasmic reticulum kinase) is a potential therapeutic strategy for these diseases. A recently published study demonstrated that oral treatment with a newly characterized PERK inhibitor was able to rescue disease phenotypes displayed in prion disease model mice. Here, we discuss the background and rationale for targeting PERK as a viable therapeutic approach as well as implications of these findings for other neurodegenerative diseases. The promise and caveats of applying this strategy for disease therapy also are discussed.

Original languageEnglish (US)
Article number30
JournalAlzheimer's Research and Therapy
Issue number3
StatePublished - May 29 2014

ASJC Scopus subject areas

  • Neurology
  • Clinical Neurology
  • Cognitive Neuroscience


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