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Gene therapy of primary T cell immunodeficiencies.

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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https://www.hal.inserm.fr/inserm-00817790
Contributor : Alain Fischer <>
Submitted on : Thursday, April 25, 2013 - 12:32:17 PM
Last modification on : Wednesday, August 19, 2020 - 11:16:37 AM
Long-term archiving on: : Friday, July 26, 2013 - 4:03:15 AM

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

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