Condition: Artemis (DCLRE1C ) Deficient Severe Combined Immunodeficiency · Sponsor: Assistance Publique - Hôpitaux de Paris
The purpose of this study is to evaluate the Safety and Efficacy of Gene Therapy of the severe combined immunodeficiency (SCID) caused by mutations in the human DCLRE1C gene (Artemis) by transplantation of a single dose of autologous CD34+ cells transduced ex vivo with the G2ARTE lentiviral vector expressing the DCLRE1C cDNA.
This description comes directly from the study's public registry record.
Marina CAVAZZANA, MD, PhD · +33 144495068 · m.cavazzana@aphp.fr
Jinmi BAEK, Master · +33 1 42 19 28 49 · jinmi.baek@aphp.fr
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| Department of Pediatric Immunology, Hematology and Rheumatology UIHR, Necker-Enfants Malades Hospital | Paris, France | Recruiting |
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Source record: clinicaltrials.gov/study/NCT05071222