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Article
Correction of B-cell development in Btk-deficient mice using lentiviral vectors with codon-optimized human BTK
X-linked agammaglobulinemia (XLA) is the most common primary immunodeficiency (PID) in man and caused by mutations in the Bruton’s tyrosine kinase (BTK) gene. XLA is characterized by a B-cell differentiation arre...
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Article
Correction of murine Rag1 deficiency by self-inactivating lentiviral vector-mediated gene transfer
Severe combined immunodeficiency (SCID) patients with an inactivating mutation in recombination activation gene 1 (RAG1) lack B and T cells due to the inability to rearrange immunoglobulin (Ig) and T-cell recepto...