New mechanism of X-linked anhidrotic ectodermal dysplasia with immunodeficiency: impairment of ubiquitin binding despite normal folding of NEMO protein

Blood. 2011 Jul 28;118(4):926-35. doi: 10.1182/blood-2010-10-315234. Epub 2011 May 26.

Abstract

Nuclear factor-κB essential modulator (NEMO), the regulatory subunit of the IκB kinase complex, is a critical component of the NF-κB pathway. Hypomorphic mutations in the X-linked human NEMO gene cause various forms of anhidrotic ectodermal dysplasia with immunodeficiency (EDA-ID). All known X-linked EDA-ID-causing mutations impair NEMO protein expression, folding, or both. We describe here 2 EDA-ID-causing missense mutations that affect the same residue in the CC2-LZ domain (D311N and D311G) that do not impair NEMO production or folding. Structural studies based on pull-down experiments showed a defect in noncovalent interaction with K63-linked and linear polyubiquitin chains for these mutant proteins. Functional studies on the patients' cells showed an impairment of the classic NF-κB signaling pathways after activation of 2 NEMO ubiquitin-binding-dependent receptors, the TNF and IL-1β receptors, and in the CD40-dependent NF-κB pathway. We report the first human NEMO mutations responsible for X-linked EDA-ID found to affect the polyubiquitin binding of NEMO rather than its expression and folding. These experiments demonstrate that the binding of human NEMO to polyubiquitin is essential for NF-κB activation. They also demonstrate that the normal expression and folding of NEMO do not exclude a pathogenic role for NEMO mutations in patients with EDA-ID.

Publication types

  • Case Reports
  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Blotting, Western
  • Ectodermal Dysplasia 1, Anhidrotic / genetics*
  • Ectodermal Dysplasia 1, Anhidrotic / metabolism
  • Enzyme Activation / genetics
  • Female
  • Humans
  • I-kappa B Kinase / genetics*
  • I-kappa B Kinase / metabolism
  • Immunologic Deficiency Syndromes / genetics*
  • Immunologic Deficiency Syndromes / metabolism
  • Male
  • Mutation, Missense
  • NF-kappa B / metabolism
  • Pedigree
  • Protein Binding
  • Protein Folding
  • Signal Transduction / genetics
  • Ubiquitin / metabolism*
  • Young Adult

Substances

  • IKBKG protein, human
  • NF-kappa B
  • Ubiquitin
  • I-kappa B Kinase