A defect in the nuclear translocation of CIITA causes a form of type II bare lymphocyte syndrome

Immunity. 1999 Feb;10(2):163-71. doi: 10.1016/s1074-7613(00)80017-5.

Abstract

The severe immunodeficiency type II bare lymphocyte syndrome (BLS) lacks class II MHC gene transcription. One defect from a complementation group A type II BLS patient is a 24 aa deletion in the MHC class II transactivator (CIITA). We show here that the molecular defect present in this protein is a failure of CIITA to undergo nuclear translocation. This defect was mapped to a position-dependent, novel nuclear localization sequence that cannot be functionally replaced by a classical NLS. Fusion of this 5 aa motif to an unrelated protein leads to nuclear translocation. Furthermore, this motif is not critical for transactivation function. This is a description of a genetic disease resulting from a novel defect in the subcellular localization of a transcriptional coactivator.

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Amino Acid Sequence
  • Animals
  • COS Cells
  • Cells, Cultured
  • Fluorescent Antibody Technique
  • Genes, MHC Class II*
  • Humans
  • Molecular Sequence Data
  • Nuclear Proteins*
  • Severe Combined Immunodeficiency / genetics*
  • Trans-Activators / physiology*
  • Transcription, Genetic
  • Transfection
  • Translocation, Genetic*

Substances

  • MHC class II transactivator protein
  • Nuclear Proteins
  • Trans-Activators