Amsterdam UMC, Universitair Medische Centra

Developing a curative gene therapy for Crigler-Najjar syndrome.
The CureCN project has ended.

The Tytgat Institute for Liver and Intestinal Research is one of the research institutes of the Amsterdam University Medical Centers, Academic Medical Center at the University of Amsterdam, Amsterdam Gastroenterology and Metabolism (AMC) of the University of Amsterdam. The AMC, the first Academic Medical Center in the Netherlands, was the result of the merge of two hospitals in Amsterdam with the Medical Faculty of the University of Amsterdam. The three main tasks of the AMC are: 1) patient care - it is a tertiary referral center, 2) medical research - both basic and translational, and 3) education for medical students, nurses, medical biology students etc.

The liver and intestinal research of the AMC is concentrated in the Tytgat Institute. Housing more than 70 basic and clinical scientists, we are one of the leading international research centers in gastroenterology and hepatology. Research in the Tytgat Institute is both fundamental and translational, but always built on a clinical question, providing results that can be translated into clinical practice. Extensive collaboration between physicians and researchers ensures a close link between the clinic and basic research.

We are exploring the use of viral vectors as novel treatment for liver and intestinal diseases, and were the first to report a proof of concept study showing the feasibility of gene therapy to treat Crigler-Najjar syndrome using adeno-associated virus (AAV). In the CureCN project, the Tytgat Institute/AMC will be responsible for the inclusion of CN patients in the trial and for establishing a World Registry of CN patients, to increase knowledge on the natural course of this disease. The Tytgat Institute will also be participating in pre-clinical studies in juvenile rats to enable AAV treatment early in life and to allow effective vector re-administration.

Staff members involved

  • Photo of
    Piter Bosma
    Team Leader & WP4 Leader
  • Photo of
    Sem Aronson
    Deputy Team Leader & WP3 Leader
  • Photo of
    Ulrich Beuers
    Deputy Team Leader & Deputy WP Leader
Amsterdam UMC, Universitair Medische Centra logo
Amsterdam UMC, Universitair Medische Centra
Tytgat Institute
Meibergdreef 69
105BK Amsterdam
the Netherlands