Gene therapy of severe combined immunodeficiencies.
The concept that the outcome of a devastating disease can be modified by inserting a transgene into abnormal cells is appealing. However, the gene-transfer technologies that are available at present have limited the success of gene therapy so far. Nevertheless, severe combined immunodeficiencies are a useful model, because gene transfer can confer a selective advantage to transduced cells. In this way, a proof of concept for gene therapy has been provided.
Fischer
Alain
A
INSERM U429, Hôpital Necker, 149 rue de Sèvres, 75015 Paris, France. fischer@necker.fr
Hacein-Bey
Salima
S
Cavazzana-Calvo
Marina
M
D000243:Adenosine Deaminase / Q000235:genetics; D000818:Animals; D003573:Cytochrome b Group / Q000235:genetics; D015316:Genetic Therapy; D006801:Humans; D016511:Severe Combined Immunodeficiency / Q000235:genetics* / Q000276:immunology / Q000628:therapy*; D013601:T-Lymphocytes
D003573:Cytochrome b Group; D000243:Adenosine Deaminase