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  • Title: Gene therapy of primary T cell immunodeficiencies.
    Author: Fischer A, Hacein-Bey-Abina S, Cavazzana-Calvo M.
    Journal: Gene; 2013 Aug 10; 525(2):170-3. PubMed ID: 23583799.
    Abstract:
    Gene therapy of severe combined immunodeficiencies has been proven to be effective to provide sustained correction of the T cell immunodeficiencies. This has been achieved for 2 forms of SCID, i.e SCID-X1 (γc deficiency) and adenosine deaminase deficiency. Occurrence of gene toxicity generated by integration of first generation retroviral vectors, as observed in the SCID-X1 trials has led to replace these vectors by self inactivated (SIN) retro(or lenti) viruses that may provide equivalent efficacy with a better safety profile. Results of ongoing clinical studies in SCID as well as in other primary immunodeficiencies, such as the Wiskott Aldrich syndrome, will be thus very informative.
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