In vitro and in vivo model systems for human diseases modeling

The course aims to provide students with an in-depth understanding of the use of experimental disease models in biomedical research. It will explore the rationale behind using animal and patient-derived models, their contributions to scientific advancements, and the ethical and regulatory considerations associated with their use.

Through this course, students will:
· Gain a historical and conceptual understanding of animal models, with a focus on genetically engineered mice and their role in biomedical research.
· Understand the application of transgenic mouse models, including knock-in, knock-out, and CRISPR/Cas9-mediated genome editing techniques.
· Analyze the role of mouse models in cancer research, from tumor initiation to preclinical drug evaluation.
· Explore patient-derived experimental disease models, including the development and applications of induced pluripotent stem cells (iPSCs).
· Study the use of mouse models for neuromuscular diseases, metabolic disorders (such as diabetes and obesity), and immunological conditions (autoimmune diseases and immunodeficiencies).
· Examine the pathogenetic mechanisms of diseases in animal models, including the role of the microbiota in immunity, metabolism, and inflammatory conditions.
· Learn about cutting-edge approaches in patient-derived disease modeling, such as 3D organoids, organ-on-chip technology, and AI-driven personalized medicine strategies.
· Acquire a comprehensive understanding of ethical considerations and regulatory guidelines, including the 3R principles (Replacement, Reduction, and Refinement) in animal research.

By the end of the course, students will be equipped with the necessary knowledge to critically assess and apply disease models in biomedical research, contributing to the development of innovative therapies and personalized medicine.

At the end of the course, students will have acquired a comprehensive understanding of experimental disease models in biomedical research. The learning outcomes include knowledge acquisition, practical application, critical thinking, communication skills, and independent learning abilities.

1. Knowledge acquisition
Students will:
· Understand the historical and scientific rationale behind the use of animal models in biomedical research, including their strengths and limitations.
· Gain in-depth knowledge of genetically engineered mouse models, including transgenic, knock-in, knock-out, and CRISPR/Cas9-based genome editing techniques.
· Learn about the application of mouse models in cancer research, neurodegenerative diseases, metabolic disorders, and immunological conditions.
· Understand the significance of patient-derived experimental disease models, including iPSCs, 3D organoids, and organ-on-chip technologies.
· Explore ethical considerations and regulatory guidelines, including the 3R principles and legislation governing the use of animal models in research.

2. Application of knowledge and understanding:
Students will:
· Apply their knowledge to design and critically evaluate experimental disease models for preclinical research.
· Analyze the advantages and limitations of different modeling approaches in addressing specific biomedical questions.
· Comprehend how metabolic/microbiota profiling and preclinical drug testing strategies can be used to study disease progression and therapeutic responses.
· Interpret and integrate multi-omics data into patient-derived models to identify disease-specific biomarkers and therapeutic targets.
· Consider ethical and regulatory aspects when designing and implementing in vivo and in vitro experiments.


3. Critical thinking
Students will:
· Develop the ability to critically assess the validity and applicability of animal and patient-derived models in biomedical research.
· Evaluate the ethical implications of using experimental models, balancing scientific progress with animal welfare concerns.
· Formulate independent opinions on the future directions of disease modeling, including emerging technologies and alternative approaches.

4. Communication skills
Students will:
· Be able to clearly communicate complex biomedical concepts related to experimental disease models to both specialized and non-specialized audiences.
· Develop the ability to present research findings effectively through oral presentations.
· Engage in scientific discussions, demonstrating an ability to interpret, critique, and synthesize relevant literature.

5. Learning abilities
Students will:

· Acquire autonomous learning skills, enabling them to stay updated on new advancements in biomedical research and experimental modeling.
· Develop a multidisciplinary approach, integrating knowledge from genetics, oncology, immunology, microbiology, neurology, stem cell biology and bioengineering.
· Enhance their ability to adapt to evolving research methodologies, including the integration of artificial intelligence and machine learning in personalized medicine.

By achieving these learning outcomes, students will be well-prepared to contribute to biomedical research, with competencies that support careers in academia, industry, and translational medicine.