Gene therapy for immunodeficiency diseases.
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.
Fischer
Alain
A
INSERM U 429, Hôpital Necker-Enfants Malades, Paris, France. alain.fischer@nck.ap-hop-paris.fr
Hacein-Bey-Abina
Salima
S
Cavazzana-Calvo
Marina
M
D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D007153:Immunologic Deficiency Syndromes / Q000628:therapy*; D016896:Treatment Outcome