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Personal profile

Research interests

My research focuses on the development of advanced molecular therapies for rare (neurometabolic) diseases, with a particular emphasis on in vivo gene editing. In addition to technological innovation, I address implementation challenges to improve the availability, accessibility, and affordability of innovative therapies, enabling timely clinical translation and patient access.

Specialisation

Assistant Professor Molecular Therapy Development | Emma Center for Personalized Medicine | Department of Human Genetics | Amsterdam UMC

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Collaborations and top research areas from the last five years

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