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ASGCT 2021 | T cell and HSC gene editing for the treatment of Hyper-IgM Type 1

Valentina Vavassori, PhD, IRCCS San Raffaele Scientific Institute & Vita-Salute San Raffaele University, Milan, Italy, describes a gene editing approach under investigation for the treatment of hyper IgM Type 1 (HIGM1), an immunodeficiency disorder caused by mutations in the gene for CD40L, a T-cell surface molecule. The strategy involves the insertion of a 5’-truncated corrective CD40L cDNA into the native gene, which restores gene function and maintains the physiological regulation of the gene. This editing strategy was applied to both autologous T cells and hematopoietic stem cells (HSCs), which were then transplanted into mouse models to compare therapeutic efficacy of the two cell types. This interview took place during the American Society for Cell & Gene Therapy 24th Annual Meeting 2021.