AAV vector gene therapy for the leukodystrophy vanishing white matter

Research in the Amsterdam Leukodystrophy Center (Department of Pediatrics) aims at improving insight into leukodystrophies and developing treatment. One such leukodystrophy is vanishing white matter (VWM), an inherited disease caused by mutations in any of the five genes encoding subunits of the translation initiation factor eIF2B. It is a devastating disease with chronic neurological deterioration starting in early childhood. VWM disease can be fatal in early life and there is no curative therapy available.

This project aims to investigate the potential of AAV gene therapy in preclinical proof-of-concept studies. You will work on AAV vector design and test functionality of gene therapy vectors in disease models and human post mortem organotypic slices. The application will be tested for efficacy and safety in animal models, that resemble the human disease pathology. The project also entails to develop disease- and vector-specific tests for vector characterization to support future clinical translation.

The project is embedded within the gene therapy group of Dr. Niek van Til focusing on state-of-the-art AAV gene therapy towards a curative therapy for VWM.

You will:

• Test efficacy and safety of AAV vectors for treatment in animal disease models for VWM;
• Drive the development of in vitro systems to test potency of AAV vectors;
• Assay development to characterize AAV vectors;
• Work collaboratively with scientists within the department and externally;
• Keep up to date with current literature in the field;
• Present data cross-functionally within the departments, at conferences and publish scientific data.

We are looking for an enthusiastic PhD student, who:

• Has acquired a Master’s degree in the field of biotechnology/gene therapy, neurosciences, molecular biology or equivalent;
• Has experience with designing, quantification and testing functionality of AAV vectors;
• Holds an Article 9 qualification for animal experimentation;
• Is fluent in English;
• Is self-motivated and can work independently, but also functions well within a team environment;
• Has attention to detail, excellent documentation, communication and presentation skills.