Gene therapy for severe combined immunodeficiency.

Marina Cavazzana-Calvo1, Chantal Lagresle, Salima Hacein-Bey-Abina

  • 1Département de Biothérapie, Hôpital Necker Enfants Malades, 75015 Paris, France. m.cavazzana@nck.ap-hop-paris.fr

Annual Review of Medicine
|January 22, 2005
PubMed
Summary

Severe combined immunodeficiency (SCID) involves T cell differentiation blocks, leading to fatal immune deficiencies. Recent advances offer molecular therapies beyond stem cell transplants.

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