A Research Fellow position is immediately available under the supervision of Michela Milani, Project Leader in the group led by Alessio Cantore 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/liver-gene-therapy.html)
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 biotechnology 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 >140 patients and led to the filing and approval of 2 novel advanced therapy medicines.
The position. We are looking for a motivated Research Fellow to work on an exciting new project aiming at developing an innovative in vivo lentiviral vector gene therapy strategy for hemophilia A. In particular, we will develop different lentiviral vectors encoding an engineered version of human factor VIII (FVIII) transgene under the control of different megakaryocyte-specific promoters. These vectors will be tested in vitro and in vivo in hemophilia A mouse models to select the best candidate to achieve effective FVIII expression in platelets after in vivo gene therapy to hematopoietic stem and progenitor cells. This project stems from our solid and extensive experience in in vivo gene therapy for hemophilia and our recent discovery of a window of opportunity in newborns to target in vivo hematopoietic stem and progenitor cells by intravenously administered lentiviral vectors.
Main tasks
- Molecular cloning of new LV constructs targeting FVIII expression to megakaryocytes
- Production and in vitro characterization of recombinant lentiviral vectors
- In vivo testing in mouse models of hemophilia A of the generated LV
- Immunological assays to evaluate immunogenicity of the developed strategy
- Data analysis and critical interpretation of the results
- Discussion of project results at internal and external meetings
Qualifications and skills
- Degree in biology/biotechnology (top grades) and research experience in competitive laboratories
- The selected candidate is expected to apply for a Ph.D. program in the future
- Cell culture and molecular biology experience
- Previous experience working with mice is considered a plus
- High accuracy, reliability, precision
- High motivation to work in an international environment
- Excellent organizational skills
- Friendly, collaborative, pro-active mindset
- Good oral and written English skills
We offer
- 1-year renewable fixed-term contract (assunzione a tempo determinato)
- RAL 25.000 €
- Opportunity to learn new cutting-edge advanced techniques
- A dynamic environment and the chance to give an essential contribution to the clinical translation of innovative ATMP
Applications, including a CV and the names and contact details of at least one referee, should be sent by email to milani.michela@hsr.it.
Evaluation of applications/interviews will begin immediately and continue until the position is filled. The selected candidate is expected to start their role in January 2027.
Research Fellow • Milano, Italia
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