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

Abstract

X-linked hyper-IgM syndrome is characterized by absent IgG, IgA, and IgE with normal/elevated IgM due to defects in the CD40 ligand gene essential for immunoglobulin class switch recombination. Previous studies using gene therapy in murine models resulted in abnormal lymphoproliferation due to unregulated expression of the gene. Targeted genome modification may be a safe and effective alternative providing definitive treatment.

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