In vitro and in vivo model systems for human diseases modeling

A.Y. 2024/2025
6
Max ECTS
42
Overall hours
SSD
BIO/11 BIO/13 BIO/14 MED/04
Language
English
Learning objectives
The purpose of this course is to train the students in the field of innovative models for human diseases ranging from in vitro to in vivo models. The course will emphasize the strengths, limitations, and applications of each model system, as well as their relevance to translational research and clinical practice.

The course is divided into two distinct modules:
1. Creating in vivo models of human diseases
2. Creating patient-derived experimental disease models
Expected learning outcomes
At the end of the course, students are expected to acquire knowledge of the experimental disease models in biomedical research and the advantages and pitfalls of each approach. Students will learn the principles, methodologies, applications, and pitfalls of the presented models, and will gain a better understanding of the ethical challenges associated with their applications.
Single course

This course can be attended as a single course.

Course syllabus and organization

Single session

Responsible
Lesson period
Third trimester
Course syllabus
1. Creating in vivo models of human diseases
Generation of transgenic mice and murine models for neurodegenerative metabolic, immune disease and cancer
This course provides an in-depth exploration of the use of mouse models in human diseases with particular emphasis on the contribution of selected model systems to the study of neurodegenerative, metabolic and immune diseases and cancer. Technical advancements in imaging and monitoring techniques as well as high-throughput phenotyping approaches will also be presented. Finally, the advantages and the pitfalls of every system will be thoroughly considered discussing how a "multiple system approach" is increasingly, becoming the approach of choice to gain a better understanding of the pathogenic mechanism underlying these diseases.

The main topics will be:
I. Historical perspective on the use of mouse models in biomedical research
II. Genetically engineered animals: definition of a transgenic mouse (knockin, Knockout, inducible and constitutive models, CRISPR/Cas9-mediated genome editing). Reasons why the use of animal models is critical for scientific research and problems associated with animal experimentation.
III. Mouse models of neurodegenerative diseases: behavioral assays for studying cognitive function and motor deficits; therapeutic interventions and drug testing in neurology mouse models
IV. Modeling Metabolic Diseases: metabolic syndrome; metabolic phenotyping techniques; targeting metabolic pathways for therapeutic development
V. Modeling Cancer: transgenic and xenograft mouse models of cancer; Tumor initiation, progression, and metastasis in mouse models; Preclinical evaluation of anticancer therapeutics
VI. Modeling Immunological Diseases: autoimmune disorders; cancer immune contexture in mice; immunomodulatory therapies in mouse models
VII. Ethical considerations and regulatory guidelines including legislation and 3R principles.

2. Creating patient-derived experimental disease models
This course provides an in-depth exploration of patient-derived experimental disease models, focusing on their significance in biomedical research, drug discovery, and personalized medicine. Students will examine various types of patient-derived models, their applications and generation methods. The course will also address the challenges and future directions in utilizing these models for understanding disease mechanisms and developing therapeutic interventions. Ethical considerations associated with the use of patient-derived models will also be discussed.

The main topics will be:
I. Introduction to Patient-Derived Models: overview of patient-derived experimental disease models, historical perspective and significance in biomedical research
II. Generation of 3D organoid models; generation of 3D organoids with increased functional complexity; 3D cultures of tissue explants;
III. Genetic manipulation: CRISPR/Cas9 editing, large-scale perturbation tools
IV. Applications of Patient-Derived Models : recapitulating disease phenotypes, mechanisms, and progression; drug screening, personalized medicine approaches; identification of disease-specific markers and therapeutic targets; preclinical studies:
V. Future Directions and Innovations: advancements in patient-derived modeling technologies (organ-on-chip models); integration of multi-omics data in model development and analysis; Application of artificial intelligence and machine learning in personalized medicine
VI. Ethical guidelines for obtaining patient samples; Data management and protection in patient-derived research
Prerequisites for admission
No prior knowledge is required
Teaching methods
Lectures will be held in presence. Participation is mandatory.
Teaching Resources
Slides of the lectures and updated published articles will be shared.
Assessment methods and Criteria
The exam will be oral.
Applied biology
BIO/13 - EXPERIMENTAL BIOLOGY - University credits: 1
Lessons: 7 hours
Professor: Grassi Fabio Maria
Experimental medicine and pathophysiology
MED/04 - EXPERIMENTAL MEDICINE AND PATHOPHYSIOLOGY - University credits: 3
Lessons: 21 hours
Molecular biology
BIO/11 - MOLECULAR BIOLOGY - University credits: 1
Lessons: 7 hours
Pharmacology
BIO/14 - PHARMACOLOGY - University credits: 1
Lessons: 7 hours
Professor: De Palma Clara