Abstract

Definitive treatment for CD40 Ligand defects are currently only achieved with hematopoietic stem cell transplant. As HSCT carries significant risks, there is a clear need for improved therapeutic modalities. Previous studies using CD40L-/- bone marrow corrected by retroviral-vector transfer of CD40L cDNA in mice resulted in abnormal lymphoproliferation. An alternative approach is targeted gene repair using TALENs (Transcription Activator-Like Effector Nucleases) or CRISPRs (Clustered Regularly Interspaced Short Palindromic Repeats), which target specific DNA sequences and create double-strand breaks, combined with homologous donor sequences serving as repair templates.

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