Familial Hemophagocytic Lymphohistiocytosis Type
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CLINICAL_TRIALS research · 2024-12-16Clinical Trial Update: Safety and Efficacy of Gene Therapy of FHL Type 3 Caused by Mutations in the Human UNC13D Gene by Transplantation of a Single Dose of Autologous CD34+ Cells Transduced Ex Vivo with the UNC13D LV Vector Expressing the UNC13D CDNA [Phase 1, Phase 2]
A Phase 1/2 clinical trial is evaluating the safety and efficacy of autologous CD34+ cells transduced with a UNC13D lentiviral vector to tre…