PhD student

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Position for PhD student

on gene therapy for lysosomal storage diseases

The laboratory of Molecular Stem Cell Biology1 at the Center for Lysosomal and Metabolic Diseases2, Departments of Clinical Genetics3 and Pediatrics4 of the Erasmus University Medical Center in Rotterdam, The Netherlands, embedded within the research school MGC (Medical Genetics Centre South West Netherlands and the Erasmus MC Graduate school5,6, is searching for a highly motivated candidate to join our group to develop lentiviral gene therapy for lysosomal storage diseases, to study the molecular mechanisms involved, and to promote clinical implementation.

1. https://www.erasmusmc.nl/en/sophia/research/groups/molecular-stem-cell-biology-of-lysosomal-storage-diseases

2. https://www.erasmusmc.nl/en/sophia/research/group/lysosomal-and-metabolic-diseases-clinical-research

3. https://www.erasmusmc.nl/research/departments/clinical-genetics

4. https://www.erasmusmc.nl/en/sophia/patient-care

5. http://www.medgencentre.com/

6. https://www.eur.nl/en/erasmusmc/graduate-school

Our research

Lysosomal storage diseases are a group of ~50 genetic diseases in which one of the lysosomal enzymes responsible for degradation of macromolecules is defective. This results in accumulation of substrates in lysosomes, lysosomal pathology, and eventually cell death. Patients with a lysosomal storage disease have symptoms in multiple tissues and organs including the central nervous system, muscle, cartilage, bone, and visceral organs. We have developed hematopoietic stem cell-mediated lentiviral gene therapy for lysosomal storage diseases in animal models. We are now optimizing this therapy in preparation for a clinical trial and we are investigating the molecular mechanisms by which pathology is restored, with a focus on the central nervous system and passage of the blood-brain barrier.

We use a variety of techniques in the field of molecular biology, biochemistry, cell biology and pathology, including lentiviral gene transduction, bone marrow transplantations, immunofluorescent analysis, functional analysis of mobility and cognition, and genome-wide expression analysis including quantitative proteomic analysis. Our research has strong links with the clinic, in which patients are treated and assessed, and we founded the startup company LentiCure (https://lenticure.nl) to bring lentiviral gene therapy to patients.

Your qualifications

A background in molecular biology/cell biology is required, but specific experience with the techniques mentioned is not. We seek a candidate with a MSc with an interest in molecular medicine, and who is enthusiastic, ambitious, a good team player and has a good level of speaking and writing English.

Our offer

We offer a position for 4 years (PhD student) according to the gross monthly salary amounts to a maximum of € 3939,- based on a full-time working week of 36 hours. Furthermore, you are entitled to a holiday allowance of 8% of your salary, an end-of-year bonus of 8.3% of your annual salary, reimbursement of travel expenses and a personal development budget. The terms of employment are in accordance with the Collective Bargaining Agreement for University Medical Centers (CAO UMC).

Literature

1. Liang et al, PMID: 36284764

2. Liang et al PMID: 35662813

3. Liang et al PMID: 38288553

4. Catalano et al PMID: 38033460

5. Catalano et al PMID: 38085235

To apply:

Please send a letter of motivation, your CV, and the names and e-mail addresses of three referees that can be contacted directly to Prof. Dr. W.W.M. Pim Pijnappel at w.pijnappel@erasmusmc.nl

Soort dienstverband: Fulltime

Salaris: Tot €3.939,00 per maand

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