Personal profile
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
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Bilirubin, the Gold Within
Seppen, J. & Bosma, P., 2012, In: Circulation. 126, 22, p. 2547-2549Research output: Contribution to journal › Editorial › Academic › peer-review
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Disruption of HNF1α binding site causes inherited severe unconjugated hyperbilirubinemia
van Dijk, R., Mayayo-Peralta, I., Aronson, S. J., Kattentidt-Mouravieva, A. A., van der Mark, V. A., de Knegt, R., Oruc, N., Beuers, U. & Bosma, P. J., 2015, In: Journal of hepatology. 63, 6, p. 1525-1529Research output: Contribution to journal › Article › Academic › peer-review
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Gene replacement therapy for genetic hepatocellular jaundice
van Dijk, R., Beuers, U. & Bosma, P. J., 2015, In: Clinical reviews in allergy & immunology. 48, 2-3, p. 243-253Research output: Contribution to journal › Review article › Academic › peer-review
Open AccessFile23 Downloads (Pure) -
ATP11C targets basolateral bile salt transporter proteins in mouse central hepatocytes
de Waart, D. R., Naik, J., Utsunomiya, K. S., Duijst, S., Ho-Mok, K., Bolier, A. R., Hiralall, J., Bull, L. N., Bosma, P. J., Oude Elferink, R. P. J. & Paulusma, C. C., 2016, In: Hepatology (Baltimore, Md.). 64, 1, p. 161-174Research output: Contribution to journal › Article › Academic › peer-review
Open AccessFile11 Downloads (Pure) -
A metabolite-based resistance mechanism against malaria
Figueiredo, A., Rastogi, S. T., Ramos, S., Nogueira, F. T., de Villiers, K., Gonçalves de Sousa, A. G., Votborg-Novél, L., von Wedel, C. C., Tober-Lau, P., Jentho, E., Pagnotta, S., Mesquita, M., Cardoso, S., Bortolussi, G., Muro, A. F., Tranfield, E. M., Thibaud, J., Duarte, D., Sousa, A. L. & Pinto, S. N. & 17 others, , 12 Jun 2025, In: Science. 388, 6752, eadq6741.Research output: Contribution to journal › Article › Academic › peer-review
Open AccessFile37 Downloads (Pure)