PhD Defence Lieke Baas

Short description thesis:

Since the emergence of gene therapy research in the 1990s, people with hemophilia and their healthcare providers have hoped that this innovation might one day provide a cure. However, gene therapy also raises ethical questions. Therefore, this thesis scrutinizes the meaning of ‘cure’ and investigates how gene therapy for hemophilia can be further developed and implemented in an ethically responsible manner, responsive to the needs of stakeholders.

The analysis demonstrates that “cure” can refer to normalization of the body, obtaining a normal life, or change in narrative identity. Given these variations, the term should be used cautiously and context-specifically to prevent misunderstanding and unrealistic expectations.

I end with three concrete recommendations for the further development of gene therapy and hemophilia care in general: to reflect on the goals of gene therapy, to have attention for the ethics of communicating about gene therapy and to design care for hemophilia in a way that can enhance the independence of people living with hemophilia.