Biotechnologie

Course goals

Upon successful completion of this course, students will be able to:

• demonstrate knowledge of the fundamental principles of human genetics;
• apply bioinformatics techniques to analyse and interpret genomic data;
• understand the basic principles of the various techniques used to investigate diseases at the molecular level;
• provide pharmacotherapeutic recommendations based on a patient’s genetic profile;
• explain how companion diagnostics are used to select the most appropriate therapy;
• evaluate the opportunities and limitations of gene therapy;
• assess the social, ethical, and societal implications of genome analysis, gene therapy, and personalized medicine;
• design and experimentally validate a guide RNA (gRNA) for genome-editing purposes;
• engage in academic discussion using sound argumentation, active listening, and effective discussion leadership;
• critically reflect on scientific issues and consider multiple perspectives.

Content

Molecular medicine is a rapidly evolving field focused on understanding the molecular basis of disease and developing innovative therapeutic approaches.
Throughout this course, students will explore the fundamental concepts of molecular biology. The course examines how genetic variation contributes to disease development and how molecular techniques are used to identify and study such variation. In addition, recent advances in genome sequencing and genome-editing technologies, including CRISPR-Cas9, will be discussed, with particular attention to their potential impact on the future of medicine.
The course is structured around four key themes central to molecular medicine: technology, diagnostics, treatment, and societal impact. A major focus of the course is understanding the interrelationship between these themes in the context of diseases such as cancer, diabetes, and neurodegenerative disorders. Through the analysis of case studies, students will learn how molecular diagnostics and personalized medicine are applied to develop tailored treatments for individual patients.
In addition to theoretical knowledge, the course includes practical laboratory training. Students will gain hands-on experience with CRISPR-Cas9 technology. Using bioinformatics tools, students will design a guide RNA, which will subsequently be tested experimentally in the laboratory for functional validation. Reporter cells will be used to demonstrate successful genome modification within cellular DNA. These practical sessions are designed to provide students with a deeper understanding of genome-editing techniques that have received considerable scientific and societal attention in recent years.
By the end of the course, students will have developed an advanced understanding of the molecular basis of disease and the cutting-edge technologies used in their diagnosis and treatment. The course aims to inspire students and equip them with the knowledge and skills necessary to contribute to future advances in medicine.