Safety and Efficacy Study of Transplantation of Autologous CD34+ Cells Transduced With the G2ARTE Lentiviral Vector Expressing the DCLRE1C cDNA in Artemis (DCLRE1C) Deficient Severe Combined Immunodeficiency Patients (ARTEGENE)
- Age
- Up to 47 Months
- Sex
- Any
- Trial phase
- Phase 1/2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 7 (estimated)
- Sponsor
- Assistance Publique - Hôpitaux de Paris · Other
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Check Your EligibilityThe 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.
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