Seminars in Hematology
Volume 41, Issue 4 , Pages 272-278, October 2004

Gene therapy for immunodeficiency diseases

  • Alain Fischer

      Affiliations

    • INSERM U 429, Hôpital Necker-Enfants Malades, Paris, France
    • Corresponding Author InformationAddress correspondence to Alain Fischer, MD, INSERM U 429, Hôpital Necker-Enfants Malades, 149 rue de Sèvres-75015 Paris, France
    • ,
  • Salima Hacein-Bey-Abina

      Affiliations

    • INSERM U 429, Hôpital Necker-Enfants Malades, Paris, France
    • ,
  • Marina Cavazzana-Calvo

      Affiliations

    • INSERM U 429, Hôpital Necker-Enfants Malades, Paris, France

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.

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PII: S0037-1963(04)00126-X

doi:10.1053/j.seminhematol.2004.07.003

Seminars in Hematology
Volume 41, Issue 4 , Pages 272-278, October 2004

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