PhD or Postdoctoral Research position 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 and to study the molecular mechanisms involved.

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. We collaborate with Leiden University Medical Center and with various national and international partners and are member of the European Reference Networks EURO-NMD and metabERN.

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

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 or PhD degree 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. Candidates applying as Post-Doctoral researcher should have at least one manuscript published or in submission in an international peer-reviewed journal.

Erasmus MC stands for a healthy population and excellence in healthcare. By conducting groundbreaking work, we aim to push boundaries through leading the way in research, education and healthcare. We have access to the latest equipment and techniques in a state-of-the-art environment.

The project will be performed within the research group Molecular Stem Cell Biology at the Departments of Pediatrics and Clinical Genetics of the Erasmus MC, which are part of the Center for Lysosomal and Metabolic Diseases, a multidisciplinary center for patient treatment, clinical and basic/applied research.