Table 1.
Ongoing clinical trials of gene therapy using autologous HSCs to treat inherited disorders in pediatric patients.
| Disease | Approach | Gene |
Clinical study (clinicaltrials.gov) |
|---|---|---|---|
| SCID-X1 | LV | IL2RG | (6) NCT03311503 NCT03601286 |
| ADA-SCID | LV | ADA | (16) NCT02999984 NCT02022696 NCT01852071 NCT03765632 |
| Wiskott–Aldrich Syndrome | LV | WAS | (29, 30) NCT01347242 NCT01410825 NCT03837483 |
| X-linked chronic granulomatous disease (CGD) | LV | Gp91phox |
NCT01855685 NCT02757911 NCT02234934 |
| Leucocyte adhesion deficiency (LAD) | LV | CD18 |
NCT03812263 NCT03825783 |
| SCID due ARTEMIS defect | LV | DCLRE1C | NCT03538899 |
| Transfusion dependent β-thalassemia | LV | HBB | (31, 32) NCT03207009 NCT02906202 |
| Transfusion-dependent β-thalassemia | GE | HBB | NCT03728322 |
| Sickle cell disease | LV | HBB | (33) NCT02140554 NCT03282656 |
| Fanconi anemia | LV | FANCA | NCT03157804 |
| Metachromatic leukodystrophy (MLD) | LV | ARSA | (34) |
| X-Adrenoleukodystrophy (ALD) | LV | ABCD1 | (35) |
| Mucopolysaccharosidosis type I | LV | IDUA | NCT03488394 |
LV, lentiviral vector; GE, gene editing.