IL-7 receptor deficient SCID with a unique intronic mutation and post-transplant autoimmunity due to chronic GVHD

Clin Immunol. 2007 Nov;125(2):159-64. doi: 10.1016/j.clim.2007.06.007. Epub 2007 Sep 12.

Abstract

Severe combined immunodeficiency (SCID) may result from a variety of genetic defects that impair the development of T cells. Signaling mediated by the cytokine interleukin-7 is essential for the differentiation of T cells from lymphoid progenitors, and mutations of either the interleukin-7 receptor alpha chain (IL-7Ralpha) or its associated cytokine receptor chain, the common gamma chain (gammac), result in SCID. Here we report a case of SCID due to heterozygous mutations of the IL7R gene encoding IL-7Ralpha. A previously unrecognized mutation found within intron 3 created a new exon between exons 3 and 4 in the mRNA transcribed from this allele, producing a truncated, unstable mRNA. This mutation illustrates the necessity of evaluating both coding and non-coding regions of genes when searching for pathogenic mutations. Following hematopoietic stem cell transplantation of our patient, immune reconstitution was accompanied by two unusual complications, immune-mediated myositis and myasthenia gravis.

Publication types

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

MeSH terms

  • Base Sequence
  • Graft vs Host Disease / genetics
  • Graft vs Host Disease / immunology*
  • Hematopoietic Stem Cell Transplantation
  • Humans
  • Infant
  • Introns
  • Male
  • Myasthenia Gravis / immunology
  • Myositis / immunology
  • Point Mutation
  • Receptors, Interleukin-7 / genetics*
  • Receptors, Interleukin-7 / immunology
  • Severe Combined Immunodeficiency / genetics*
  • Severe Combined Immunodeficiency / immunology
  • Severe Combined Immunodeficiency / therapy

Substances

  • Receptors, Interleukin-7
  • interleukin-7 receptor, alpha chain