Gene therapy of primary T cell immunodeficiencies. - Inserm - Institut national de la santé et de la recherche médicale Access content directly
Journal Articles Gene Year : 2013

Gene therapy of primary T cell immunodeficiencies.


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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inserm-00817790 , version 1 (25-04-2013)



Alain Fischer, Salima Hacein-Bey-Abina, Marina Cavazzana-Calvo. Gene therapy of primary T cell immunodeficiencies.. Gene, 2013, 525 (2), pp.170-173. ⟨10.1016/j.gene.2013.03.092⟩. ⟨inserm-00817790⟩
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