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Gene therapy of primary T cell immunodeficiencies |
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| Authors: | Alain Fischer Salima Hacein-Bey-Abina Marina Cavazzana-Calvo |
| Institution: | 1. INSERM U768, Paris, France;2. Paris Cité, Université Paris Descartes, Imagine Institute, Paris, France;3. Immunology and Pediatric Hematology Department, Assistance Publique-Hôpitaux de Paris, Paris, France;4. Biotherapy Department, Necker Children''s Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France;5. Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, Assistance Publique-Hôpitaux de Paris, INSERM, Paris, France |
| 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. |
| Keywords: | ADA adenosine deaminase GVHD graft versus host disease HSC hematopoietic stem cell HSCT hematopoietic stem cell transplantation IPS induced pluripotent cells LV Lentivirus PIDs primary immunodeficiencies RV retrovirus SAE serious adverse event SCID severe combined immunodeficiency SIN self inactivated WAS Wiskott Aldrich syndrome |
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