PhD: Gene Therapy for Leukodystrophies (AAV & Genome Editing)

SIGRA; Amsterdam; On-site; EUR 39,000 - 49,000; Full time

Job summary

Amsterdam UMC, in the POLARIS doctoral network, offers a PhD project focusing on preclinical evaluation of gene therapy for leukodystrophies using VWM and MLD mouse models. The candidate will explore AAV vectors, bridgeRNA-guided recombinases, and translational readouts under supervision by Dr Niek van Til at the Amsterdam Leukodystrophy Center (ALC).

The role provides international collaboration and planned secondments.

Qualifications

• Master's degree in life sciences or related discipline with strong research background.
• Experience in translational neuroscience or rare disease research is preferred.
• Formal certification for working with laboratory animals (e.g., FELASA) is preferred.
• Fluency in English (written and spoken) is essential.
• Eligibility criteria for MARIE SKŁODOWSKA CURIE programs apply (no prior doctoral degree).
• Experience gained during internships or research projects is relevant.

Responsibilities

• Test efficacy and safety of AAV gene therapy in VWM and MLD mouse models.
• Apply genome-editing approaches, including bridgeRNA-guided recombinases, to assess therapeutic efficacy.
• Use molecular techniques and in vivo mouse models to study disease mechanisms and treatment responses.
• Analyze and interpret experimental results; align preclinical readouts with clinically meaningful outcomes.
• Collaborate with Amsterdam UMC and POLARIS partners; participate in network training and secondments.

Skills

Molecular techniques

Viral vectors

Mouse studies

Analytical skills

Communication

Collaboration

Education

Master's degree in life sciences

Master's degree in molecular sciences

Job description

Amsterdam UMC, in the POLARIS doctoral network, offers a PhD project focusing on preclinical evaluation of gene therapy for leukodystrophies using VWM and MLD mouse models. The candidate will explore AAV vectors, bridgeRNA-guided recombinases, and translational readouts under supervision by Dr Niek van Til at the Amsterdam Leukodystrophy Center (ALC).

The role provides international collaboration and planned secondments.