Research interests

  • Gene Therapy.
    My main focus is to develop Adeno Associated liver directed gene therapy to treat inherited unconjugated hyperbilirubinemia. This research is funded by the Dutch Najjar foundation and by ZonMw. In collaboration with the group of Prof.Dr Labrunne and the AFM we plan to start a phase 1 trial end 2016 or start 2017. Succesfull application of AAV in this patients will also enable us to use this strategy to treat other inherited liver diseases such as PFIC.
  • Liver function.
    The role of inter cellular signaling in the development of liver diseases and bile formation. Using in vitro and in vivo knock-down we will determine the role of liver specific transport proteins in liver function. In addition to their role in bile formatin we are investigating their potential role in inter cellular signaling for instance via the formation of exosomes and microvesicles. 

specialisation

Liver directed gene therapy for inherited severe liver disorders

Research output

  1. Bilirubin, the Gold Within

    Research output: Contribution to journalEditorialAcademicpeer-review

  2. Disruption of HNF1α binding site causes inherited severe unconjugated hyperbilirubinemia

    Research output: Contribution to journalArticleAcademicpeer-review

  3. Gene replacement therapy for genetic hepatocellular jaundice

    Research output: Contribution to journalReview articleAcademicpeer-review

  4. ATP11C targets basolateral bile salt transporter proteins in mouse central hepatocytes

    Research output: Contribution to journalArticleAcademicpeer-review

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