04 ott - Milano
Gruppo San Donato
IRCCS San Raffaele Scientific Institute in Milan, Italy, is looking for a Research Fellow to join a newly established research line under the supervision of Michela Milani, Project Leader in the lab led by Alessio Cantore (https://tinyurl.com/muj89n8d) at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget).
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 for 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 activities:
• 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.
Who we are looking for:
• 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 in work with mice is considered a plus.
• High accuracy, reli
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