MSCA-DN PhD position in Gene Therapy for Leukodystrophies

SIGRA; Amsterdam; On-site; EUR 36,000 - 45,000; Full time

Benefits offered by this job

Living allowance provided

Mobility allowance

Pension ABP contribution

Transport reimbursement

GoodHabitz online learning

Job summary

Amsterdam UMC hosts a POLARIS doctoral candidate position focusing on preclinical gene therapy for leukodystrophies. The project investigates AAV-based approaches in VWM and MLD mouse models, employing bridgeRNA-guided recombinases and genome-editing to assess therapeutic efficacy.

Hosted within the Amsterdam Leukodystrophy Center, the PhD candidate will collaborate across international partners, benefit from network-wide training, and advance translational neuroscience toward patient-oriented

Qualifications

• Master's degree in life sciences or related discipline required.
• Strong hands-on experience with molecular techniques.
• Experience with viral vectors and/or in vivo mouse studies preferred.
• Interest in responsible animal research and robust experimental design.
• Excellent communication and collaboration in international teams.

Responsibilities

• Assess efficacy and safety of AAV gene therapies in VWM/MLD mouse models.
• Apply genome-editing approaches to test therapeutic efficacy.
• Use molecular techniques and mouse models to study disease mechanisms.
• Analyze data and align preclinical readouts with clinical outcomes.
• Collaborate with Amsterdam UMC and POLARIS partners; participate in training and secondments.

Skills

Molecular techniques

Virus vectors

Mouse models

Translational neuroscience

Scientific communication

Education

Master's degree in life sciences

Master's degree in molecular sciences

Tools

AAV vectors

Genome-editing tools

BridgeRNA-guided recombinases

Job description

Would you like to contribute to the development of gene therapies for rare neurological diseases? Join the POLARIS doctoral network and work on innovative gene therapy approaches for leukodystrophies in an international and interdisciplinary research environment.

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.

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.

We are looking for a motivated candidate with a Master's degree in life sciences, molecular sciences or a related discipline. A background in molecular sciences is particularly relevant to this project.

You have relevant research experience and an interest in translational neuroscience and rare disease research. Experience gained during internships or research projects is relevant. We are looking for:

• experience with molecular techniques;
• experience with viral vectors and/or in vivo mouse studies is an advantage;
• an interest in responsible use of animal models, animal welfare principles and robust experimental design; experience with behavioural or neurological phenotyping is an advantage;
• an analytical and accurate approach, with the ability to plan, organise and interpret experimental work;
• good communication and collaboration skills and an interest in working in an international and interdisciplinary research environment.

Formal certification for working with laboratory animals, such as FELASA or an equivalent qualification, is preferred.

Fluency in English, both written and spoken, is essential.

Eligibility criteria

Important, strict eligibility criteria according to MARIE SKLODOWSKA CURIE programs apply to applicants:

1. You must not already hold a doctoral degree.
2. At the time of the application deadline, you must have a Master's degree and must not have resided or carried out your main activity (work, studies, etc.) in the Netherlands for more than 12 months in the three years prior to the contract start date.
3. You must be able and willing to work in an international training network, participate in network-wide training activities and undertake planned secondments with POLARIS partner organisations.

A contract for 12 months, with the intention to extend for a total of 3 years. The Guideline PhD contract applies to this vacancy.

Salary scale OIO: € 3.217 to € 4.077 gross based on full-time employment (depending on education and experience) and a year-end bonus of 8.3%. Calculate your net salary here.

You will receive a living allowance of €4,010 per month, adjusted with a country-specific correction factor and subject to local employer contributions and tax regulations. In addition, candidates will receive a monthly mobility allowance of €710. If applicable, a family allowance of €660 per month is provided for fellows with a spouse and/or dependent children. Successful applicants are also eligible for parental leave in accordance with Dutch legislation.

Vacation hours: 190.4 per year based on full-time employment and possibility to save additional hours.

Free and unlimited access to our online learning environment GoodHabitz.

Pension accrual with the ABP, of which we pay 70% of the premium.

Reimbursement of your public transport expenses. Do you prefer biking to work? Then we have a good bicycle scheme.

An active staff association and Jong Amsterdam UMC association, both of which organize fun (sports) activities and events.

Non-EU/EEA/Swiss applicants require a valid residence permit to live and work in the Netherlands.

You will work at the Amsterdam Leukodystrophy Center (ALC) at Amsterdam UMC, an internationally recognised expertise center for leukodystrophies. The center combines specialist clinical care, diagnostics, patient-oriented research and translational neuroscience. Research at the ALC focuses on understanding disease mechanisms in rare white matter disorders and translating these insights into improved models, biomarkers and therapeutic strategies.

Dr Niek van Til's team focuses on advancing viral vector technologies, including adeno-associated virus (AAV)-based gene therapies and hematopoietic stem cell (HSC) gene therapy, to improve treatment of leukodystrophies such as vanishing white matter disease and metachromatic leukodystrophy (MLD). By combining expertise in gene delivery, translational research and preclinical model development, the team aims to accelerate the translation of promising therapies from the laboratory to patients.

You will also be part of the international POLARIS doctoral network. You will work with researchers from different scientific disciplines and collaborate with academic and industry partners across Europe. Through secondments, network-wide training and transferable-skills development, you will build experience beyond your own research project and develop an international research network.

POLARIS is committed to open, transparent and merit-based recruitment. We value diversity and welcome candidates with different backgrounds, perspectives and experiences.

The application deadline is 5 October 2026. We aim to select the final candidate in October/November 2026.

A reference check and screening may be part of the selection procedure. If you join Amsterdam UMC, we request a VOG (Certificate of Good Conduct) for a number of job categories.

Acquisition in response to this vacancy will not be considered.