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After decades of research, the first gene therapy treatments for hemophilia are now becoming available. These treatments offer the hope of a cure, but they also raise important ethical questions. This research explores how gene therapy for hemophilia can be developed and used in an ethically responsible way.

Two main questions guided the study. 1) What are the ethical concerns around gene therapy for hemophilia? And 2) What does it actually mean to “cure” hemophilia?

Through a review of existing literature and interviews with people with hemophilia and health care providers three key ethical themes emerged. The first theme is ‘living up to expectations’. Many people hope that gene therapy will provide a real cure, but it is still unclear whether that goal is fully achievable. The second theme is ‘independence’. For many people with hemophilia, the idea of living a more independent life is a major reason to consider gene therapy. The third theme is ‘costs and fair access’. Gene therapy is expensive, and there are concerns about who will be able to access it and how it can be distributed fairly.

The research also shows that the word “cure” can mean different things: restoring the body, living a normal life, or even changing how someone sees themselves. These meanings can all be valid, as long as we are clear about what we mean.

Moving forward, responsible gene therapy requires clear goals, honest communication about what is possible, and designing hemophilia care in a way that supports patients’ independence.