Gene Therapy for Primary Immunodeficiencies - 11/08/11
, S. Hacein-Bey-Abina, PharmD, PhD a, b, d, e, M. Cavazanna-Calvo, MD, PhD a, b, d, eRésumé |
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.
Le texte complet de cet article est disponible en PDF.Keywords : Severe combined immunodeficiencies, Gene therapy, Hematopoietic stem cells, Retrovirus, Lentivirus, Wiskott Aldrich, Chronic granulomatous disease, Clinical trial
Plan
Vol 30 - N° 2
P. 237-248 - mai 2010 Retour au numéroBienvenue sur EM-consulte, la référence des professionnels de santé.
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