MSCA-DN PhD position in Gene Therapy for Leukodystrophies

POLARIS doctoral network

POLARIS is a European Marie Skłodowska-Curie Doctoral Network focused on accelerating therapy development for leukodystrophies: rare genetic disorders affecting the brain's white matter. The network aims to strengthen the full translational pathway, from understanding disease mechanisms and developing disease models to preclinical therapy testing, clinical readiness and patient-informed treatment priorities.

To achieve this, POLARIS brings together expertise in leukodystrophy biology, advanced disease models, therapeutic innovation, biomarker development, clinical translation, patient engagement, and ethical, regulatory and health-economic aspects of therapy development. The network will train a cohort of doctoral candidates in an international and interdisciplinary environment, combining academic research, industry-based preclinical development, secondments, network-wide training and transferable-skills development.

PhD project
For this position, the recruiting organisation is Amsterdam UMC, in the Netherlands. You will be employed by Amsterdam UMC and embedded in the POLARIS consortium. Academic supervision and disease-specific expertise will be provided by Dr Niek van Til at the Amsterdam Leukodystrophy Center (ALC).

This doctoral project focuses on the preclinical assessment of gene therapy approaches in leukodystrophy mouse models for vanishing white matter disease (VWM) and metachromatic leukodystrophy (MLD).

You will investigate the efficacy and safety of AAV gene therapy in VWM and MLD mouse models. In addition, you will use genome-editing approaches, including bridgeRNA-guided recombinases, to investigate therapeutic efficacy.

The project requires relevant experience in molecular techniques and an interest in responsible and scientifically robust use of mouse models in translational research. Planned secondments will provide hands-on experience in the production of gene therapy vectors and with the Vereniging voor Innovatieve Geneesmiddelen (VIG), where you will contribute to evaluating access pathways for advanced therapy medicinal products (ATMPs).

Your academic embedding will be at the Amsterdam Leukodystrophy Center, with the anticipated PhD defence at the Faculty of Medicine, Vrije Universiteit Amsterdam.

Would you like to know more about the different phases within the PhD trajectory? You can read more about this on this page.

As a doctoral candidate, you will contribute to the preclinical development of gene therapy approaches for leukodystrophies. Your work includes:

• testing the efficacy and safety of AAV gene therapy in VWM and MLD mouse models;
• applying genome-editing approaches, including bridgeRNA-guided recombinases, to investigate therapeutic efficacy;
• using molecular techniques and in vivo mouse models to investigate disease mechanisms and treatment responses;
• analysing and interpreting experimental results and considering how preclinical readouts can be aligned with clinically meaningful outcomes;
• collaborating with colleagues at Amsterdam UMC and POLARIS partner organisations and participating in network-wide training activities and planned secondments.

You will be part of an international and interdisciplinary research environment. Throughout the PhD project, you will have opportunities to further develop your scientific and transferable skills through the POLARIS doctoral training programme.