Ateneo - UPO

Stem cells, induced pluripotent stem cells (iPSCs), cell reprogramming, cell therapy, gene therapy, gene transfer methods, animal models of genetic diseases, clinical trials

The course will refer to the scientific literature present in various online journals, such as Human Gene Therapy, Gene Therapy, Molecular Therapy, Nature, Science, Journal of Gene Medicine, ...

At the end of the course, the student will be able to critically understand articles of cell and gene therapy, will have knowledge of the bases for the development of cell and / or gene transfer methods and of the cell and gene therapy methods used in preclinical studies and clinical trials

Knowledge of cellular and molecular biology

Presentations by the teacher (ppt) and scientific articles in pdf format

Oral presentation with discussion of a scientific project

Stem cells, cellular reprogramming and use of these cells in cell therapy protocols in preclinical and potential applications in the clinic.
Isolation, in vitro expansion and preparation of cells for experimental cell therapy.
Vectors used for gene transfer: viral vectors and non-viral. Origin, construction and modi�cation.
Modulation of Expression cassettes: Constitutive and regulated expression. transcriptional or post-transcriptional expression regulation Immune response to vectors and
transgenes used in gene transfer.
Concepts of ex vivo and in vivo gene therapy.
Clinical trials using cell and gene therapy strategies of target diseases.
use of animal models to study genetic diseases

At the end of the course, the student will be able to critically understand articles of cell and gene therapy, will have knowledge of the bases for the development of cell and / or gene transfer methods and of the cell and gene therapy methods used in preclinical studies and clinical trials