Your challenge

This new research project focuses on translational research to develop state-of-the-art gene therapies for vanishing white matter (VWM) leukodystrophies. VWM is 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. Recent developments in the gene therapy field has opened the potential to translate these molecular technologies into effective treatment options by using viral vectors and gene-editing technologies. The PhD candidate will work on translational research to design and test functionality of gene therapy vectors and genome-editing technologies. These applications will be tested for efficacy and safety in both in vitro and in vivo animal models, that resemble the human disease pathology for clinical translation and towards curative therapies for VWM patients.

Key responsibilities:

  • Drive the development of in vitro testing systems that mimic VWM pathology to test gene therapy vectors;
  • Design and test potency of lentiviral vectors and adeno-associated virus (AAV) vectors for treatment of VWM disease;
  • Perform studies to test genome-editing technologies, such as based on CRISPR/Cas9 to correct VWM mutations;
  • Test these molecular applications in both in vitro and in vivo efficacy and safety studies;
  • 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.

Your profile

  • Requires 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 lentiviral vectors and/or adeno-associated virus vectors;
  • Has experience in molecular biology, cloning, CRISPR/Cas or other genome-editing technologies;
  • Has a proven record using in vitro disease models for vector functionality testing and assay development;
  • Experience with in vivo disease models mimicking human disease is preferred;
  • 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.

Your work environment

We offer a PhD position for four years in the department of Pediatrics, within the group of prof. dr. Marjo van der Knaap. The department of Pediatrics at VUmc has an internationally renowned research focus on childhood leukodystrophies. This multidisciplinary research group is composed of child neurologists, molecular and cell biologists and is supported by experienced technicians. The group is embedded within the laboratories of the Center of Neurogenomics and Cognitive Research (CNCR) of the VU University Amsterdam ( The research area mainly focuses on clinical signs, MRI/MRS, genetics, neuropathology, electrophysiology and molecular and cellular aspects of neurological diseases, and on developing potential curative therapies including gene therapy. More information on our research can be found at the website.

Working at Amsterdam UMC means working in an inspiring and professional environment in which developing one`s talents and skills is encouraged. We offer you ample opportunity for development, deepening and broadening, additional training and a place to grow!

We offer

Salary Scale PHD: 2495 tot 3196 euro gross when employed full-time (depending on qualifications and experience).

The monthly wage will increase yearly to the maximum wage in the fourth year of EUR 3.196 gross per month.

In addition we offer:
– Additional benefits include an 8.3% end-of-year bonus and 8% holiday pay.
– The opportunity to follow further training and development.
– Pension is accrued at the ABP. VUmc contributes a large part of the premium (70%).
– In addition to excellent accessibility by public transport, we reimburse 75% of your OV travel costs (commuting costs).

We offer an initial contract for 12 months that can be extended (after evaluation) for another 36 months.

For more information about our employment conditions, please visit our website.
For Dutch citizens it is mandatory to provide a VOG (Verklaring Omtrent Gedrag).

Information and application

Don’t hesitate to call us!
If you’re interested about this position, you can contact dr. Niek van Til, lecturer/team leader, via telephone number 020-4448704.
If you need more information about the application procedure, contact Brend de Jong, corporate recruiter at 020-4448704.

You can apply till 1 March 2021 by using the apply button.

Acquisition based on this vacancy is not appreciated.