Gene therapy for immunodeficiency diseases

Semin Hematol. 2004 Oct;41(4):272-8. doi: 10.1053/j.seminhematol.2004.07.003.

Abstract

Primary immunodeficiency diseases represent good targets for hematopoietic stem cell-targeted gene therapy. Severe combined immunodeficiencies (SCID) have been the first examples of successful gene therapy based on the ex vivo usage of retroviral vectors. New advances in the technology of gene transfer should further promote gene therapy as a safe and effective therapeutic strategy of immunodeficiency diseases.

Publication types

  • Review

MeSH terms

  • Gene Transfer Techniques
  • Genetic Therapy / methods*
  • Humans
  • Immunologic Deficiency Syndromes / therapy*
  • Treatment Outcome