Table 1.

Preclinical and clinical gene therapy studies for primary immune deficiencies (PID).

Preclinical studiesClinical 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 studiesClinical 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  

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