The course intends to provide students with the most recent applications of biotechology to pharmacology, with emphasis on new biotechnological drugs, drug discovery and individualization of drug therapies on the basis of patients genotype (pharmacogenetics and pharmacogenomics).
The students will learn about a selection of methodological approaches to study intracellular signalling pathways that are activated by ligand-receptor interaction in living cells, organotypic slices and entire organisms. When mastered, these techniques (in conjunction with the abilities and the competencies derived from the other courses) will allow the student to have a strong background on high-quality functional assays -based on optical imaging techniques- to study cellular responses triggered by the exposure to specific endogenous agonist or drugs. The students will learn about up-to-date background knowledge needed for the full comprehension of the rapid progress of biotechnological drugs. Students will understand the evolution of the field with an historical perspective view showing the parallel development that drug and molecular biology fields experienced until their final merging in the new discipline. The protein and nucleic acid engineering, and the application of molecular biology in the generation of innovative therapies will be considered starting from approved biotechnological drugs. Finally the students will learn about the genetic basis of drug responses, with deep insights into the use of pharmacogenetics for therapy personalization.
Lectures 1: Introduction: definition, history of biotechnology and pharmacology. Lecture 2: Main classes of biotechnological drugs, problems with the production and use of biologics, biosimilars. Lecture 3: Advanced therapies: general concepts, gene therapy: definition, vectors, current drugs in genetic diseases, oncology and future prospective in other therapeutic areas. Lecture 4- Therapeutic proteins: classifications and general concepts, hormones, fusion proteins, monoclonal antibodies, and their evolutions, immunotherapy. Lecture 5- Nucleic acids as drugs: classifications and basic concepts for antisense, ribozyme, aptamer, decoy and siRNA; focus on antisense oligonucleotides and aptamers: mechanism of action and the innovation brought by the last generation of antisense oligonucleotides. Lecture 6- Fluorescent tools for the study of receptors Lecture 7- Molecular properties and applications of fluorescent proteins Lecture 8- Use of fluorescent protein-based biosensors in monitoring ligand-receptor complex formation and downstream intracellular effects Lecture 9- Measuring intracellular Ca2+ concentrations and fluctuations in living cells and neurons Lecture 10- Drugs affecting the actin cytoskeleton dynamics 11- Genetic basis of individual drug response. 12- Effects of genetic variants on drug pharmacokinetics 13-Effects of genetic variants on drug pharmacodynamics 14- Clinical pharmacogenetics