Gene therapy of lymphoid primary immunodeficiencies.
Gene therapy offers an attractive option to the most severe forms of primary immunodeficiency diseases. Identification of disease-associated genes as well as advances in the technology of gene transfer into hematopoietic progenitor cells have set the basis for the first clinical trials. Settings characterized by the potential for a selective advantage provided to transduced cells are the first diseases to target. The recent example of successful treatment of severe combined immunodeficiency-X1 (gammac deficiency) illustrates this potential.
Fischer
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Hôpital Necker Enfants Malades, Paris, France. fischer@necker.fr
D015316:Genetic Therapy; D006801:Humans; D007153:Immunologic Deficiency Syndromes / Q000628:therapy*; D016511:Severe Combined Immunodeficiency / Q000628:therapy