Post-Doctoral position in Gene Editing for Neurodegenerative Disorders

A Post-doc fellow is immediately available in the group of Gene and Neural Stem Cell Therapy for Lysosomal Storage Diseases led by Dr. Angela Gritti at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Milan, Italy (https://research.hsr.it/en/institutes/san-raffaele-telethon-institute-for-gene-therapy/gene-and-neural-stem-cell-therapy-for-lysosomal-storage-diseases.html ).

The position. We are looking for a highly motivated and resourceful Post-doc candidate for a translational project focused on precise genetic modifications in glia cells to silence/correct disease-causing mutations in Alexander disease, an autosomal dominant neurodegenerative disorder. We already optimized novel CRISPR/Cas9-based approaches. In the project, the Postdoc will provide in vitro and in vivo proof-of-concept of efficacy and safety of these editing platforms in murine and human disease models.

The Institute. SR-Tiget was created in 1996 as a joint-venture between Fondazione Telethon and Ospedale San Raffaele (OSR), with the mission to perform cutting edge research on gene and cell therapy and to translate its results into therapeutic advances for genetic diseases. The Institute is located within the OSR campus, which includes a large multi-disciplinary research hospital, a biomedical research center, a university and also hosts several biotechnologies companies. Over the years, SR-Tiget has given a pioneering contribution to the gene and cell therapy field with relevant discoveries in vector design, gene transfer and gene editing strategies, stem cell biology, identity and mechanism of action of regulatory cells in immune responses and innate immune cells in cancer. SR-Tiget has also established the resources and framework for translating these advances into novel experimental therapies and has implemented several successful gene therapy clinical trials, which have already treated >120 patients and led to the filing and approval of 2 novel advanced therapy medicines.

Main tasks

• development of AAV and LV platforms to target astrocytes
• development of nanoparticle-based delivery systems (in collaboration with national and international teams)
• in vitro and in vivo proof-of-concept studies in murine disease models
• in vitro gene editing analyses and disease modeling studies in human hiPSC-based models

We offer

• Competitive salary depending on qualifications and experience
• A dynamic environment and the chance to benefit from professional training, mentorship, networking opportunities, and assistance in writing funding proposals to support the research project

Applications, including a CV and the names and contact details of at least two referees, should be sent by e-mail to Dr. Angela Gritti (Group Leader, [email protected] ) and Dr. Vasco Meneghini (Project Leader, [email protected] ). Evaluation of applications/interviews will begin immediately and continue until the position is filled.