Ateneo - UPO

the principle of Cell and Gene Therapy Stem cells Gene transfer by viral and non viral vectors

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, ...

The primary objective of the course is to provide the fundamentals of gene and cell therapy necessary to understand preclinical and clinical studies published in recent years. Particular emphasis is placed on the viral and non-viral vectors used in gene therapy, covering the investigation methods used to optimize gene transfer constructs, delivery modalities, and current applications. The course also addresses the assessment of potential unintended effects of gene transfer and explores the use of gene and cell therapy in various fields beyond genetic diseases alone (e.g., cardiovascular conditions, cancer).

Knowledge of cellular and molecular biology. Basic knowledge of pathology (genetic and metabolic diseases)

Power point presentations and PDF articles

Students with physical disabilities, Learning Disabilities or Special Education Needs can request specific services and tools via the Staff Sviluppo e Coordinamento Carriere e Servizi alle Studentesse e agli Studenti, consulting the University webpage: https://www.uniupo.it/en/services/services-students-physical-or-learning-disabilities

Students with disabilities, learning disabilities or special education needs, once they have contacted the University Staff, can refer to the tutor in charge of the course to define the examination modalities, concerning academic aspects.

Written test on the topics of the program, with multiple choice questions and open questions. Moreover the students in groups they have to present a poster and an article with a commentary and a seminar discussion on a cell and gene therapy paper chosen by each group

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 modification.
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.

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