[Gene therapy for immune deficiencies].
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 (gamma c deficiency) illustrates this potential.
Hôpital Necker-Enfants Malades, INSERM U429, 149 rue de Sèvres-75015 Paris.
A
A
Fischer
S
S
Hacein-Bey
F
F
Le Deist
G
G
de Saint Basile
J P
JP
de Villartay
M
M
Cavazzana-Calvo
D015316:Genetic Therapy / Q000379:methods; D006801:Humans; D007153:Immunologic Deficiency Syndromes / Q000235:genetics*