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 |