In vitro and in vivo model systems for human diseases modeling

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