SINE compounds activate exportin-1 degradation via an allosteric mechanism

Insights

Selinexor and KPT-185 are cancer drugs that target Exportin 1 (XPO1) degradation. New structures reveal these drugs act as allosteric degraders, stabilizing XPO1 for degradation by the CRL5ASB8 system.

Area of Science:

  • Molecular biology
  • Structural biology
  • Cancer research

Background:

  • Exportin 1 (XPO1/CRM1) overexpression in cancer disrupts protein localization.
  • XPO1 inhibitors like selinexor restore cargo localization by targeting XPO1 for degradation.

Purpose of the Study:

  • To elucidate the mechanism of XPO1 inhibition and degradation by selinexor/KPT-185 via the CRL5ASB8 system.
  • To determine the structural basis for inhibitor-XPO1 engagement and subsequent ubiquitination.

Main Methods:

  • Cryogenic electron microscopy (cryo-EM) to determine high-resolution structures.
  • Biochemical assays to assess protein-ligand interactions and ubiquitination.

Main Results:

  • Cryo-EM structures reveal ASB8 binding to a unique drug-induced degron on XPO1.
  • Selinexor/KPT-185 binding significantly increases XPO1-ASB8 affinity, promoting CRL5ASB8-mediated ubiquitination.
  • The mechanism is allosteric, with drugs binding XPO1 to stabilize an ASB8-binding conformation, distinct from molecular glues.

Conclusions:

  • Selinexor/KPT-185 function as allosteric degraders, not molecular glues.
  • This expands the understanding of targeted protein degradation mechanisms via the CRL5 system.

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