Molecular Therapies for Otogenetic Disorders

The research group investigates the molecular basis of otogenetic disorders using an integrated approach that combines long-read transcriptomics, proteomics, patient-derived 2D and 3D inner ear and retina models, and humanized zebrafish and mouse models. They have extensive experience in developing preclinical RNA-based therapies, with one candidate already in clinical trials. Their work also explores tissue regeneration pathways and innovative therapeutic delivery methods. A key focus is advanced phenotypic profiling to identify biomarkers that can guide and assess treatment efficacy in future clinical trials. Strongly committed to patient involvement, the group collaborates closely with affected individuals, helping shape research priorities and co-founding three Dutch patient organizations: Stichting Ushersyndroom, Stichting De Negende van, and DFNA21 Stichting.

Link to research group: Molecular therapies for otogenetic disorders​