X-linked immunodeficiencies

Curr Allergy Asthma Rep. 2004 Sep;4(5):339-48. doi: 10.1007/s11882-004-0082-5.

Abstract

Recent advances in molecular genetics have allowed identification of at least seven genes involved in X-linked immunodeficiencies. This has resulted not only in improved diagnostic possibilities but also in a better understanding of the pathophysiology of these disorders. In some cases, mutations in the same gene have been shown to cause distinct clinical and immunologic phenotypes, demonstrating a strong genotype-phenotype correlation. Identification of the molecular basis of these diseases has permitted creation of disease-specific registries, with a better characterization of the clinical and immunologic features associated with the various forms of X-linked immunodeficiencies. Additionally, gene therapy has been attempted in X-linked severe combined immune deficiency (XSCID), with clear evidence of successful correction of the pathology, and the appearance of severe adverse effects.

Publication types

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

MeSH terms

  • Agammaglobulinemia / genetics
  • CD40 Ligand
  • Ectodermal Dysplasia / genetics
  • Genetic Diseases, X-Linked / immunology*
  • Genotype
  • Humans
  • Hypergammaglobulinemia / genetics
  • Immunologic Deficiency Syndromes / genetics*
  • Lymphoproliferative Disorders / genetics
  • Mutation
  • NF-kappa B / genetics
  • Phenotype
  • Polyendocrinopathies, Autoimmune / genetics
  • Protein-Losing Enteropathies / genetics
  • Severe Combined Immunodeficiency / genetics
  • Wiskott-Aldrich Syndrome / genetics

Substances

  • NF-kappa B
  • CD40 Ligand