Preclinical and clinical gene therapy studies for primary immune deficiencies (PID).
| Preclinical studies . | Clinical studies . |
|---|---|
| CGD indicates chronic granulomatous disease; LAD, leukocyte adhesion deficiency; IPEX, Immune dysfunction, polyendocrinopathy, enteropathy, X-linked. | |
| SCID due to deficiency of | ADA-SCID |
| -RAG-1 | SCID-X1 |
| -RAG-2 | CGD |
| -Artemis | JAK3-SCID |
| -IL7R | LAD |
| -PNP | WAS |
| SAP-deficiency | |
| CD40L deficiency | |
| X-linked agammaglobulinemia | |
| IPEX | |
| Preclinical studies . | Clinical studies . |
|---|---|
| CGD indicates chronic granulomatous disease; LAD, leukocyte adhesion deficiency; IPEX, Immune dysfunction, polyendocrinopathy, enteropathy, X-linked. | |
| SCID due to deficiency of | ADA-SCID |
| -RAG-1 | SCID-X1 |
| -RAG-2 | CGD |
| -Artemis | JAK3-SCID |
| -IL7R | LAD |
| -PNP | WAS |
| SAP-deficiency | |
| CD40L deficiency | |
| X-linked agammaglobulinemia | |
| IPEX | |