The Therapeutic Potential of PTEN Modulation: Targeting Strategies from Gene to Protein

N.M. McLoughlin, C. Mueller, T.N. Grossmann

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Two decades have passed since the discovery of the tumor suppressor, PTEN. A multitude of biological functions have since been revealed, suggesting potential therapeutic applications for both PTEN activation (e.g., cancer) and inhibition (e.g., neuroregeneration). Nevertheless, PTEN's therapeutic suitability has been called into question due to its “risky” profile as a tumor suppressor. To evaluate PTEN function and its various roles in disease a number of molecules have so far been developed. However, intrinsic problems associated with phosphatase inhibition and PTEN's complex regulation via post-translational modifications hinder straightforward access to selective modulators. For this reason, central questions associated with PTEN targeting remain unanswered. In this perspective, we summarize current PTEN-targeting strategies and discuss potential approaches to modulate its functional dose, considering all stages of PTEN biogenesis from direct protein modulation to the targeting of relevant miRNAs as well as the PTEN gene and mRNA
Original languageEnglish
Pages (from-to)19-29
Number of pages11
JournalCell Chemical Biology
Issue number1
Early online date16 Nov 2017
Publication statusPublished - 18 Jan 2018


  • Neurodegenerative Diseases
  • anti-gene
  • anti-sense
  • cancer
  • gene editing
  • miR-21
  • miRNA maturation
  • new modalities
  • phosphatase
  • protein-protein interaction

VU Research Profile

  • Human Health and Life Sciences


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