Gene therapy for primary immunodeficiencies.
The concept of gene therapy emerged as a way of correcting monogenic inherited diseases by introducing a normal copy of the mutated gene into at least some of the patients' cells. Although this concept has turned out to be quite complicated to implement, it is in the field of primary immunodeficiencies (PIDs) that proof of feasibility has been undoubtedly achieved. There is now a strong rationale in support of gene therapy for at least some PIDs, as discussed in this article.
Fischer
Alain
A
Developpement Normal et Pathologique du Systeme Immunitaire, INSERM U 768, Hopital Necker, 149 rue de sevres, Paris 75015, France. alain.fischer@inserm.fr
Hacein-Bey-Abina
S
S
Cavazzana-Calvo
M
M
Cavazanna-Calvo
M
M
D056704:Adaptive Immunity; D000243:Adenosine Deaminase / Q000235:genetics*; D000818:Animals; D002986:Clinical Trials as Topic; D015316:Genetic Therapy / Q000639:trends*; D006801:Humans; D007153:Immunologic Deficiency Syndromes / Q000235:genetics* / Q000276:immunology / Q000628:therapy*; D013601:T-Lymphocytes / Q000378:metabolism* / Q000473:pathology
D000243:Adenosine Deaminase