Cell Therapy and Gene Silencing

A.Y. 2024/2025
7
Max ECTS
72
Overall hours
SSD
BIO/13 BIO/14 BIO/19
Language
English
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.
Single course

This course can be attended as a single course.

Course syllabus and organization

Single session

Lesson period
First semester
Course syllabus
The course is organized in three sections including single bench laboratory and practical activities.
The Cell-based Technology section (2 CFU) 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.
The Viral Vectors for Gene Delivery section (2 CFU) will cover the aspects relative to the use of viral gene transfer/gene therapy including viral replication and gene expression strategies, retroviral and lentiviral vectors and applications, adenoviral vectors and Adenovirus-Associated virus (AAV) vectors and relative applications, innate immune response to viral vectors, the use of viral vectors for gene editing, RNAi, epigenetic silencing and genetic vaccines.
The Pharmacological aspects of Cell Therapy and Gene Silencing section (1 CFU) will cover some examples of pharmacological use and outcomes on human diseases. In details, discussion of pathological mechanisms, pharmacological strategies and clinical trials of gene therapy (in vivo and ex vivo), gene silencing approaches (siRNA and ASO) and epidrugs.

Practical sections are organized in two parts: the first will include demonstrations on cell culture, transfection, protein production and localization (1 CFU); the second part will deal with omics application by the use of online software for the analysis and interpretations of biological implications of gene therapy approaches and exercise of clinical pharmacology (1 CFU).
Prerequisites for admission
Good knowledge of General Microbiology, Biochemistry, Molecular and Cell Biology, and basic data analysis by excel software.
Teaching methods
Frontal lecture, virtual laboratory simulations, laboratory activities and software-based laboratory for dataset analysis. Lectures attendance is strongly recommended, while laboratory attendance is mandatory.
Teaching Resources
Giacca M. "Gene Therapy"
Springer, 2010
ISBN 978-88-470-1643-9
Ian Freshney R. "Culture of Animal Cells: A Manual of Basic Technique and Specialized Applications"
John Wiley & Sons, Inc., 2010
Print ISBN:9780470528129; Online ISBN:9780470649367; DOI:10.1002/9780470649367

Lecture slides, personal notes, review and article papers, and links to selected WEB sites.
It is strongly recommended that the student will participate to the lectures and use the material provided on "Cell Therapy and Gene Silencing" myAriel webpage.
Assessment methods and Criteria
The evaluation will be based on assessing the acquired theoretical knowledge and practical skills.
The exam will evaluate:
- the ability of the student to reach course objectives in terms of knowledge and understanding (multiple choices);
- the ability of the student to apply knowledge and understanding (laboratory practical reports);
- the ability of the student to use the correct terminology and to discuss the different topics in a clear and logical way (open questions).
The exam will consist in:
- reports of the single bench laboratory / practical activities (4 points cell based-technology and 4 points Pharmacological aspects of cell therapy and gene silencing);
- a written test with multiple choices and open questions (25 points)
BIO/13 - EXPERIMENTAL BIOLOGY - University credits: 3
BIO/14 - PHARMACOLOGY - University credits: 2
BIO/19 - MICROBIOLOGY - University credits: 2
Single bench laboratory practical: 32 hours
Lessons: 40 hours
Professor(s)
Reception:
By appointment
Reception:
DiSFeB - via Balzaretti 9 - piano 4
Reception:
on appointment
Reception:
by appointment
INGM, via Francesco Sforza 35 o DiSFeB, via Balzaretti 9, Milano