Table 2 Overview of current available clinical trials for different PIDs
From: Treating primary immunodeficiencies with defects in NK cells: from stem cell therapy to gene editing
Disease | Mutated gene/protein | Vector/target cell | Conditioning | Clinical trial reference |
|---|---|---|---|---|
CNKD1 | GATA2 | Allogeneic HSCT | Busulfan/fludarabine/cyclophosphamide/TBI | NCT01861106 |
WAS | WAS | SIN-LV/BM/PBSCs | RIC busulfan/fludarabine | NCT01515462 |
NCT01347346 | ||||
NCT01347242 | ||||
NCT01410825 | ||||
SIN-LV/PBSCs | None | NCT03837483 | ||
X-SCID | IL2RG | SIN-γRV/BM | None | NCT01410019 |
NCT01129544 | ||||
NCT01175239 | ||||
SIN-LV/PBSCs | Busulfan 6 mg/kg | NCT01306019 | ||
SIN-LV/BM | Busulfan 6 mg/kg | NCT01512888 | ||
ADA-SCID | ADA | SIN-LV/BM/PBSCs | Busulfan 5 mg/kg | NCT01380990 |
SIN-LV/BM/PBSCs | Busulfan 4 mg/kg | NCT01852071 | ||
NCT02022696 |