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.

• A flying start to your career in scientific research.
• Plenty of room for your drive to shape tomorrow's healthcare.
• Working on large-scale and in-house research, with motivated colleagues.
• You will be employed by Amsterdam UMC Research BV.
• A contract for 12 months, with the intention to extend for a total of 4 years. The Guideline PhD contract applies to this vacancy.
• Salary scale OIO: € 3.017 to € 3.824 gross based on full-time employment (depending on education and experience) and a year-end bonus of 8.3%. Calculate your net salary here.
• Holiday hours: 190,4 per year for fulltime and a possibility to save additional hours.
• Pension accrual with BeFrank, a modern, comprehensible and fairly priced pension.
• For >7 km each way, 100% reimbursement for public transport travel costs and, for private transport, €0.18 per km up to a maximum of 40 km each way.
• Do you prefer walking or cycling? Take advantage of our good bike scheme. Moreover, you will receive a reimbursement of €0.18 per km.

Watch this video with more information about joining Amsterdam UMC Research BV.

The Amsterdam Leukodystrophy Center offers an excellent research environment. It consists of a multidisciplinary team of clinicians and laboratory researchers focusing on many different aspects of leukodystrophies. The team contains child neurologists, 6 research PI’s, trial coordinators, research technicians, PhD students and numerous interns. Some of us work on clinical aspects of leukodystrophies using patient registries or on clinical trials in patients. Others work in the lab and execute studies on disease mechanisms, with the aim to better understand these diseases and find openings for treatment, such as developing potential curative therapies including gene therapy. Working in this multidisciplinary team means that you will gain insight into many different aspects of leukodystrophies and preclinical studies.

Amsterdam UMC Research BV supports non-profit scientific research. In doing so, we provide researchers with everything they need to excel. Our principal investigators (PIs) and project leaders offer support in the field of project management, finance and human resources. In medical scientific research projects, legal support is also provided.

Watch the video to find out more.

During the publication period, applications will be handled continuously. If the vacancy is filled, it will be closed prematurely.

If you have any questions about this position, please feel free to contact Dr. Niek van Til, via n.p.vantil@amsterdamumc.nl.

For more information about the application procedure, please contact Chey Edwards, Recruitment advisor, via c.i.edwards@amsterdamumc.nl.

A reference check, screening and hiring test may be part of the procedure. Read here whether that applies to you. If you join us, we ask you for a VOG (Certificate of Good Conduct).

Internal candidates will be given priority over external candidates in case of equal suitability.

Acquisition in response to this vacancy is not appreciated.