The PhD student will work on gene therapy development for Gyrate atrophy of the choroid and retina (GACR), a rare genetic (neuro)metabolic eye disorder. Based on disease pathology, patient’s needs, ethical considerations, therapeutic developmental time lines, and current state of the art of gene therapy, we recently posed that the most optimal therapeutic strategy is systemic treatment with a (repurposed) drug/supplement, combined with specific ocular gene therapy (Bergen and coworkers, 2023).
The candidate will use our unique set of human iPSC derived retinal GACR models and perform tailored therapeutic screens with repurposed medicine taken from the parallel
SYMPATHIC consortium (Karnebeek and coworkers 2023). The candidate will, in parallel, explore CRISPR and AAV technologies to develop new ocular gene therapy.
We have all multidisciplinary facilities, technology and expertise needed directly at our disposal. The candidate will build on the extensive experience and infrastructure of the Ophthalmogenetics lab with iPSC technology for retinal cell/organoid differentiation combined with the innovative 3D cellular bioprinting, to develop and use retinal models in pre-clinical setting.
- You will work together with other PhD students, postdocs, staff members and assistant professors of the Ophthalmogenetics lab and Emma Center for Personalized Medicine and together you look for connections between various scientific. Your project will inform and strengthen other projects, and vice versa. You contribute to the implementation of the four counters of the Emma CPM; diagnostics, therapy, care and education;
- You will contribute to education by guiding students and/or PhD candidates, assist in developing education for healthcare professionals, patients (and their loved ones) and other stakeholders, and translate new scientific insights into educational and other knowledge-sharing methods;
- You will actively participate in educational activities within Amsterdam UMC;
- Your studies will result in peer reviewed scientific publications, and will be presented at national and international conferences.