PhD in Gene Therapy for Rare 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

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