PhD position in Gene Therapy for human genetic diseases
5 giorni fa
Department of Women's and Children's Health
Organisation/Company: Università degli Studi di Padova
Department: Department of Women's and Children's Health
Research Field: Biological sciences » Biological engineering, Medical sciences » Health sciences
Researcher Profile: First Stage Researcher (R1)
Positions: PhD Positions
Country: Italy
Application Deadline: 8 Jun 2025 - 00:00 (Europe/Rome)
Type of Contract: Temporary
Job Status: Full-time
Hours Per Week: 40
Offer Starting Date: 1 Oct 2025
Is the job funded through the EU Research Framework Programme? Not funded by a EU programme
Is the Job related to staff position within a Research Infrastructure? No
Offer DescriptionPhD position in Gene Therapy - Dr. POLETTI laboratory
Employer: University of Padova (Padua, Italy)
PhD Course in: DEVELOPMENTAL MEDICINE AND HEALTH PLANNING SCIENCES
Curriculum: ONCOHEMATOLOGY AND HUMAN GENETICS, RARE DISEASES AND PREDICTIVE MEDICINE;
About the University of PadovaThe University of Padova is one of Europe's oldest and most prestigious universities. Founded in 1222, UNIPD was the home of many illustrious people who changed the cultural and scientific history of humanity, from Copernicus to Vesalius, Galileo Galilei, and from Giovan Battista Morgagni to William Harvey. UNIPD offers a wide range of PhD Programs covering hard sciences, humanities, interdisciplinary programs, life sciences, medical and biomedical sciences. To apply for a PhD Course, applicants must meet the requirements listed in the call and pass an entrance examination. All information (requirements, deadlines, selection procedure) will be available in the calls for admission.
About the PhD programThe PhD Course in Developmental Medicine and Health Planning Sciences, organized by the Department of Women's and Children's Health, aims at developing academic researchers, researchers with responsibility for research groups and biomedical laboratories, heads of clinical studies, epidemiologists, and health planners who can be inserted into research groups that deal with health planning and programming. The laboratories are located within the Pediatric Research Institute (IRP), a large facility (over 17,500 m2) equipped with state-of-the-art facilities and technologies, and a stimulating multidiscipline environment.
About the scientific projectPhD project: "Molecular evolution of a hyperfunctional β-galactosidase for the HSC Gene Therapy of GM1 Gangliosidosis". Ex vivo gene therapy (GT) is based on the transfer of a therapeutic gene into patients' hematopoietic stem cells (HSCs) for the potential cure of a genetic disease. GT is already an approved therapy in Europe and the USA (Libmeldy/Lenmeldy) for a genetic disease due to lysosomal dysfunction, i.e., metachromatic leukodystrophy, and it is showing clinical efficacy for other similar diseases. The aim of this project is the development of a GT strategy aimed at correcting the symptoms of a disease caused by β-galactosidase (β-gal) deficiency, i.e., GM1 Gangliosidosis. Our GT is based on the design of a novel, synthetic hyperfunctional human β-gal enzyme delivered to the HSCs by a last-generation lentiviral vector. The efficacy of the GT will be tested in patient-derived cells and brain organoids. If successful, this project may represent the first step to the development of an efficacious therapy for GM1 Gangliosidosis, and set a new modality for the development of novel therapies for orphan and lethal diseases.
Application RequirementsMSc in Biotechnology, Biology, Pharmacology, or Life Sciences.
Applications should include a CV, a cover letter, and the names and contact information of two references.
Early applications are highly encouraged, as the applications will be processed upon reception.
Where to applyE-mail: valentina.poletti@unipd.it
RequirementsResearch Field: Biological sciences » Biology
Education Level: Master Degree or equivalent
Research Field: Technology » Biotechnology
Education Level: Master Degree or equivalent
Research Field: Technology » Pharmaceutical technology
Education Level: Master Degree or equivalent
Skills/Qualifications: MSc in Biotechnology, Biology, Pharmacology, or Life Sciences.
Languages: ENGLISH Level Good
Research Field: Biological sciences » Biology, Technology » Biotechnology, Medical sciences » Health sciences
Additional InformationSelection process: To apply for a PhD Course, applicants must meet the requirements listed in the call and pass an entrance examination. All information (requirements, deadlines, selection procedure) will be available in the calls for admission.
Applications should include a CV, a cover letter, and the names and contact information of two references.
Early applications are highly encouraged, as the applications will be processed upon reception.
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