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Are you interested in developing innovative therapies for genetic diseases? We are looking for an enthusiastic and ambitious PhD student to complement our team on preclinical therapy development for rare metabolic disorders. Based on recently discovered novel metabolic mechanisms in our group, we are developing antisense oligonucleotide strategies to restore the metabolic balance in advanced patient-derived in vitro model systems. We make use of induced pluripotent stem cell derived neuronal models from patients and obtained via genome editing. The project is part of a larger European consortium called RADICAL-CDG.
Within RADICAL-CDG, you will work together with researchers in our team and with international colleagues on advancing the current lead compounds to a preclinical proof-of-concept stage. The focus group of genetic disease will be the Congenital Disorders of Glycosylation. Your specific role will be to optimize antisense oligonucleotides for established metabolic enzymes and investigate the timing and dosing of the metabolic and electrophysiological effects in iPSC derived neuronal models. You will work together with researchers and experts on metabolic diseases and metabolomics and will be trained on translational drug development aspects.
Tasks:
At Radboudumc, you will work in a highly collaborative, interdisciplinary, and international environment. We are a leading European center for genomic medicine and rare disease research, with strong connections to clinicians, patients, and European initiatives such as ERDERA. What sets us apart is our unique position at the interface of fundamental research on metabolomics and advanced model systems and translational research on (genetic) treatments involving clinicians and patients. This ensures that your work will have clinical relevance and impact.
You will join a group that performs preclinical research on genetic metabolic disorders aiming to improve diagnostics, understand disease mechanisms and develop treatments. Our team consists of senior scientists, technicians and PhD students and post-docs with multidisciplinary expertise of metabolomics, iPSC based disease models, therapeutics and molecular genetics.
The position is based at Radboudumc in Nijmegen, the Netherlands. Radboudumc is internationally renowned for its research and diagnostics on a variety of genetic disorders and the impact of personalized medicine. The position is primarly housed in the department of Human Genetics with a strong collaboration with Pediatrics. Both departments offer an exciting working environment with enthusiastic people from many parts of the world, providing ample critical mass and a stimulating, collaborative atmosphere for high quality research.
You bring curiosity, creativity, and energy to the table. You are proactive and take initiative, and highly value collaboration with team members and other people within our department. You are motivated, communicative, hard-working, and adaptable, while keeping your eye on the team's goals.
Your ideal profile:



