Summary of the clinical experience of hematopoietic stem cell (HSC)-gene therapy for SCID-X1.
| Study . | No. pts treated . | Longest follow-up . | Efficacy . | Toxicity . |
|---|---|---|---|---|
| GOSH indicates Great Ormond Street Hospital. | ||||
| Hôpital Necker22,25 | 10 | 10 y | Yes | T-cell leukemia (4 pts) |
| GOSH 23,24 | 10 | 7 y | Yes | T-cell leukemia (1 pt) |
| GOSH, Necker,27 NIH26 | 5 (age 10–20) | 3 y for pts with engraftment | No, improvement in 1 pt | No |
| Study . | No. pts treated . | Longest follow-up . | Efficacy . | Toxicity . |
|---|---|---|---|---|
| GOSH indicates Great Ormond Street Hospital. | ||||
| Hôpital Necker22,25 | 10 | 10 y | Yes | T-cell leukemia (4 pts) |
| GOSH 23,24 | 10 | 7 y | Yes | T-cell leukemia (1 pt) |
| GOSH, Necker,27 NIH26 | 5 (age 10–20) | 3 y for pts with engraftment | No, improvement in 1 pt | No |