PhD position in Gene Therapy for human genetic diseases - GM1 Gangliosidosis

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 Description

PhD 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 Padova

The 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 program

The 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 project

PhD 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 Requirements

MSc 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 apply

E-mail: valentina.poletti@unipd.it

Requirements

Research 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 Information

Selection 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.