03May 2025
Gene Therapy in Hemophilia A: Innovation with Safety Concerns

Gene Therapy in Hemophilia A: Innovation with Safety Concerns

Novel gene therapy sparks hope—but raises safety questions. Srivastava et al.’s trial using CD68-driven lentiviral expression of porcine factor VIII in autologous CD34+ cells marks a major step in hemophilia A research. However, overexpression in CD68+ monocyte–macrophage cells may increase thrombosis risk, especially during inflammation, and could provoke immune responses due to antigen-presenting cell involvement. Ongoing trials should closely monitor thrombotic and immunologic complications.

  • #family health

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