Sintesi dell’opportunità
This MSCA‑DN PhD offers a 3‑year fixed‑term contract at Amsterdam UMC, with a 12‑month initial period and potential extension. The project focuses on developing CRISPR‑based gene‑editing strategies and lipid‑nanoparticle delivery for two rare leukodystrophies—vanishing white matter disease and metachromatic leukodystrophy—at the University of Zurich. Candidates should hold a Master’s in life sciences, have experience in genome editing, nucleic‑acid delivery, and cellular or animal models, and be fluent in English. The role includes secondments at Amsterdam UMC and InnoSer, access to the POLARIS doctoral network, and opportunities for translational research and professional development.
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Descrizione del progetto
About your role
You will develop gene-editing strategies for two leukodystrophies: vanishing white matter disease (VWM) and metachromatic leukodystrophy (MLD). You will work at the interface of genome editing, targeted delivery and preclinical disease modelling, with the aim of advancing new therapeutic approaches.
Your research will focus on:
- Developing gene-editing strategies for VWM and MLD, including precise gene correction and whole-gene insertion.
- Developing targeted delivery approaches using lipid nanoparticles (LNPs) to reach the relevant therapeutic target cells: glial cells in the brain for VWM and hematopoietic stem and progenitor cells for MLD.
- Evaluating the efficacy, durability and safety of different editing approaches in relevant cellular and preclinical disease models.
- Assessing potential safety risks, including off-target genomic integration and long-term tolerability.
- Working across research environments through planned secondments at Amsterdam UMC and InnoSer. At Amsterdam UMC, you will gain experience with phenotypic assays in VWM and MLD models, while at InnoSer you will work on the phenotyping of leukodystrophy mouse models. Your academic research will be embedded within the Institute of Pharmacology and Toxicology at the University of Zurich, under the supervision of Prof. Gerald Schwank. The anticipated PhD defence will take place at the University of Zurich.
About you
We are looking for a motivated candidate who is interested in therapeutic genome editing, rare-disease research and translational science. You bring: - A Master’s degree in molecular biology, biotechnology, biomedical sciences, genetics, bioengineering or a related life-sciences field.
- Experience in molecular biology, genome editing, nucleic-acid delivery, mammalian cell culture or hematopoietic stem and progenitor cells, gained through internships or research projects.
- Experience with CRISPR-based editing, cloning or sequencing-based analysis of editing outcomes is advantageous.
- Experience with lipid nanoparticles or viral-vector delivery is advantageous.
- Experience with animal models, animal-welfare principles and robust experimental design is advantageous.
- A strong interest in developing new therapeutic approaches for rare genetic diseases.
- An accurate and reproducible way of working, with a strong eye for detail and the ability to plan and organise complex experimental work.
- An analytical mindset, good communication skills and the ability to collaborate across different research environments.
- The ability and willingness to work in an interdisciplinary and international research environment, collaborating with colleagues at UZH and across the POLARIS consortium.Fluency in English, both written and spoken, is essential.Eligibility criteria Important, strict eligibility criteria according to MARIE SKLODOWSKA CURIE programs apply to applicants:
- You must not already hold a doctoral degree.
- 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 Switzerland for more than 12 months in the three years prior to the contract start date.
- 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.
Our offer - A contract for 12 months, with the intention to extend for a total of 3 years;
- Salary, mobility allowance and benefits according to the applicable MSCA Doctoral Network and host-organisation rules. More detailed information about the salary will follow; ;
- Embedding in the international POLARIS doctoral training network;
- Access to network-wide training activities, secondments and transferable-skills development;
- A challenging and translational research project at the interface of genome editing, nucleic-acid delivery, rare-disease biology and preclinical therapeutic development;
- A collaborative and entrepreneurial working environment;
- An open and supportive team culture with opportunities for scientific and professional development.
Amsterdam University Medical Center
Amsterdam, Netherlands