MUNC13.4-CD34

MUNC13.4-CD34 is an ex vivo gene therapy consisting of autologous CD34+ hematopoietic stem cells (HSCs) transduced with a lentiviral vector carrying a functional human UNC13D gene. Developed by Assistance Publique - Hôpitaux de Paris (AP-HP), this therapy is specifically designed to treat familial hemophagocytic lymphohistiocytosis type 3 (FHL3). FHL3 is a rare, life-threatening immune disorder caused by mutations in the UNC13D gene, which lead to a deficiency in the Munc13-4 protein. This protein is essential for the priming and fusion of lytic granules in cytotoxic T lymphocytes (CTLs) and natural killer (NK) cells. By restoring Munc13-4 expression in the hematopoietic lineage, the therapy aims to normalize the degranulation capacity of immune cells, thereby preventing the uncontrolled immune activation and hyperinflammatory cytokine storm characteristic of the disease.

Other names

UNC13D gene therapyUNC-13D gene therapyUNC 13D gene therapyMunc13.4 gene therapyMunc-13.4 gene therapyMunc 13.4 gene therapyLentiviral vector-transduced autologous CD34+ cells encoding UNC13D