Gene and Cell Therapy

Responsabili del curriculum:

The goal of the curriculum is to train young researchers in the field of Advanced Therapy Medicinal Products (ATMPs), including gene, cell and immuno- therapies for the treatment of genetic and acquired diseases, with a specific focus on the translational aspects and the clinical/biotechnological applicability of the results.
In particular, the pathologies under study include:

  • primary immunodeficiencies
  • metabolic diseases
  • hemoglobinopathies
  • hematological malignancies and solid tumors
  • neurodegenerative diseases
  • pathologies of the neuromuscular system
  • autoimmune and auto inflammatory diseases

The development of these therapies requires a complex path, ranging from the identification and study of the genes and/or cells involved in the aetiology and pathology of the diseases investigated, the development of new therapeutic strategies and their validation in vitro and in animal models of disease, up to clinical trials in patients.
The Curriculum offers training in gene and cell therapy strategies, technologies for gene transfer and targeted genome and epigenome editing, procedures for the manipulation, engineering and transplantation of somatic stem cells and derivatives of pluripotent stem cells, the integration between experimental and computational approaches applied to the study of genetic pathologies, the main regulatory requirements for preclinical and clinical testing.

Training activities

In addition to the training program of the PhD Course in Molecular Medicine (link), PhD students will be requested to attend courses and seminars on topics of specific interest for the Currilculum (such as principles of gene and cell therapy, elements of bioethics and regulatory requirements for ATMP research and clinical trials, principles of GLP - Good Laboratory Practice).
PhD students will also have the opportunity to attend at least one international conference a year, which will allow them to present their results and participate in the debate of the gene and cell therapy community.
The Curriculum is supported by Fondazione Telethon, in line with its mission to promote biomedical research on genetic diseases, and benefits from the active contribution of the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), an internationally recognized center of excellence in the field of gene and cell therapy.

 

List of participating laboratories (A.Y. 2020/2021)