Gene therapy for inherited immunodeficiencies

Curr Hematol Rep. 2003 Jul;2(4):328-34.

Abstract

Extensive research into gene therapy technologies has produced few clinically relevant results. Advances in the understanding of the genetics of inherited immunologic diseases, gene transfer methodologies, and stem cell manipulation have all contributed to successes in treating X-linked severe combined immunodeficiency and adenosine deaminase deficiency. This review examines the progress and the problems that have arisen, and discusses the improvement and future of gene therapy for primary immunodeficiencies.

Publication types

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

MeSH terms

  • Adenosine Deaminase / deficiency
  • Genetic Therapy / methods*
  • Hematopoietic Stem Cells
  • Humans
  • Immunologic Deficiency Syndromes / pathology
  • Immunologic Deficiency Syndromes / therapy*
  • Leukopoiesis
  • Severe Combined Immunodeficiency / pathology
  • Severe Combined Immunodeficiency / therapy

Substances

  • Adenosine Deaminase