Base-edited autologous HSPCs and T cells (NIAID)

Base-edited autologous HSPCs and T cells (NIAID) is an experimental cell-based gene therapy designed to treat CD40L-HyperIgM syndrome (X-linked Hyper-IgM syndrome). The therapy involves the ex vivo genetic correction of a patient's own hematopoietic stem and progenitor cells (HSPCs) and T cells using CRISPR-based base editing technology. This process specifically targets and repairs mutations in the CD40LG gene, which encodes the CD40 ligand (CD40L) protein. In patients with this disorder, defective CD40L on activated T cells prevents B cells from undergoing antibody class switching, leading to severe immunodeficiency. By restoring functional CD40L expression, the therapy aims to re-establish normal immune signaling and antibody production. The treatment protocol includes myeloid conditioning followed by the infusion of corrected HSPCs for long-term engraftment and corrected T cells for immediate immune reconstitution.

Other names

Base-edited hematopoietic stem and progenitor cellsBase-edited T lymphocyte cellsBase-edited HSPC and T Cell Gene Therapy for CD40L-HyperIgM Syndrome