Cell therapy and gene silencing

A.Y. 2020/2021
Overall hours
BIO/13 BIO/14 BIO/19
Learning objectives
The course is aimed at providing the basis to understand the use of cell therapy and gene silencing for the treatment of human diseases. Specifically, the course will discuss the origin and heterogeneity of available human cell-based medical products and their complexity. The differences between autologous or allogenic, stem or induced pluripotent stem cells will be discussed. Further, the course will offer an overview on technologies crucial for gene and cell therapies such as gene editing and safe viral vectors for gene delivery and as basis for prophylactic and therapeutic vaccines.
Expected learning outcomes
At the end of the course, the student should possess the specific knowledge to understand the generation and the application of cell therapy and gene silencing techniques to treat human diseases. In particular, the student should have acquired the basic principles of gene therapy, including gene transfer technologies, therapeutic strategies and their efficiency. The student should also demonstrate to possess the appropriate biological terminology to communicate the most relevant and recent topics in the field of cell therapy and gene silencing.
Course syllabus and organization

Single session

Lesson period
First semester
The lessons will be held in attendance or through Microsoft Teams, Zoom Meeting or other platforms in asynchronous or synchronous transmission.
The program and the teaching material will be the same.
The exam will be oral using Microsoft Teams, Zoom Meeting or other platforms; if allowed by the emergency rules, it might be in presence as oral.
Course syllabus
The module of Cell-based Technology will cover the aspects relative to the use of cellular models in biomedical research. These include aspects of cell culture manipulation, differences between immortalised and primary cells, stems cells, iPSCs,CAR T cells, methods of transfection and protein production.
Introduction to viral gene transfer/gene therapy
Viral replication and gene expression strategies
Retroviral and lentiviral vectors and applications
Adenoviral vectors and applications
Adenovirus-Associated virus (AAV) vectors and applications
Innate immune response to viral vectors
Use of viral vectors for gene editing, RNAi and epigenetic silencing
Use of viral vectors for genetic vaccines
Prerequisites for admission
Good knowledge of General Microbiology, Biochemistry, Molecular and Cell Biology
Teaching methods
Lectures, power-point slides as well as recorded videos of the lectures
Teaching Resources
Giacca M. "Gene Therapy"
Springer, 2010
ISBN 978-88-470-1643-9
Lecture slides, personal notes and review papers. Links to selected microbiology and/or virology WEB sites and papers will be provided.
It is highly recommended that the student will participate to the lectures and use the deposited material on Ariel to prepare for the exam.sites and papers will be provided.
Assessment methods and Criteria
The exam will consist in a presentation of a scientific article relative to the topics of the Course. The evaluation will be based on assessing the acquired theoretical knowledge and presentation skills.
The exam will evaluate:
- The ability of the student to reach course objectives in terms of knowledge and understanding
- The ability of the student to apply knowledge and understanding
- The ability of the student to use the correct terminology and to discuss the different topics in a clear and logical way
BIO/13 - EXPERIMENTAL BIOLOGY - University credits: 0
BIO/14 - PHARMACOLOGY - University credits: 0
BIO/19 - MICROBIOLOGY - University credits: 0
Single bench laboratory practical: 32 hours
Lessons: 40 hours