Gene-corrected regulatory T cell therapy for IL2RA deficiency

Individuals with genetic deficiency of the alpha chain of the IL-2 receptor (IL2RA or CD25) have defective regulatory T cells (Treg). These individuals develop a range of autoimmune conditions. We demonstrate a method for ex-vivo correction of a mutated allele of IL2RA using CRISPR technology. We developed a GMP-compatible method to manufacture an autologous gene-corrected Treg product that could be used treat patients with IL2RA deficiency.