Targeted Gene Therapy in the Treatment of X-Linked Hyper-Igm Syndrome

Abstract

Background: X-linked hyper-IgM syndrome (XHIM) is characterized by absent IgG, IgA, and IgE with normal or elevated IgM due to defects in the CD40 ligand (CD40L) gene essential for B cell class switch recombination. Previous studies using gene therapy in mouse models resulted in abnormal lymphoproliferation due to unregulated expression of the CD40L gene.

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