Assistant Professor
Strategic program(s):
Biography
It is my mission to make gene therapies more efficient, specific and safe. I use advanced cellular disease models in combination with state-of-the art genome editing, molecular biology, and genomics technologies to address several key questions that are crucial for the development of gene therapy for CF, such as:
- What is the minimal amount of delivery efficiency that is required to reach clinical efficacy and is this similar for different organs?
- Which cell types take-up CF gene therapies after administration?
- Do CFTR modulators synergize with gene therapies?
- Which cell types contribute to the effectiveness of CF gene therapies?
- Can we change the tropism of therapeutic particles towards CF-relevant cell types?
- Can gene therapies be designed to target multiple organs affected by CF?
Organoids are extremely useful to address these questions, for several reasons: (1) for all of the organs affected by CF, human organoid models have been developed, (2) organoids contain disease relevant cell types and recapitulate important aspects of the CF phenotype, (3) the forskolin-induced-swelling-assay, which was developed at our institute by professor Jeffrey Beekman, enables the rapid assessment of CFTR function and determine patient-specific responsiveness towards therapies.
With my research I contribute to the development of the next generation of gene therapies, so that people suffering from Cystic Fibrosis and other genetic diseases can lead long and fulfilling lives.
