The objective of this course is to provide knowledge on: i) the path leading to drug development (basic research, pre-clinical trials and clinical studies); ii) molecular and cellular mechanisms underlying drugs activity; iii) pharmacogenetics and pharmacogenomics; iv) gene therapy; v) new molecular medications (RNA drugs); vi) role of stem cells in drug discovery and therapy. The course will also provide in addition to theoretical content a practical approach to the topics, with a specific focus on the design of experimental protocols and the use of models.
Expected learning outcomes
At the end of this course, the student will know how new molecules with therapeutic activity can be developed and will have knowledge about the molecular mechanisms underlying drug properties.
Drug development. R&D from the Nineties to present. Rational approach to drug design. Molecular modelling. Hit identification. Lead optimisation and development of medications. Preclinical development of medications and clinical studies. GMP production.
Receptors, signal transduction and receptor classes. Modulation of receptor responses. Channel receptors, G-protein coupled receptors, growth factor receptors and cytokines. Intracellular calcium homeostasis. Pharmacological modulation of post-translational modifications.
Proteolysis. Mechanisms and pharmacology of intra-and extra-cellular proteolysis.
Intercellular communication. Pharmacological regulation of synaptic function. Pharmacology of neurosecretion. Pharmacological modulation of excitatory and inhibitory synaptic transmission.
Pharmacology of gene transcription. Epigenetic drugs. RNA Drugs: Mechanisms of action, administration, chemical structure and toxicology.
Pharmacogenetics and Pharmacogenomics. The genome project and its relevance to drug development. Genomic medicine, precision Medicine, personalized medicine.
Gene therapy. Vectors and strategies of gene transfer. Gene therapy for monogenic diseases, diabetes, cancer and cardiovascular system.
Stem cells in drug development and discovery. Stem cells therapies.
Prerequisites for admission
The student must possess basic concepts of general and advanced pharmacology, biochemistry and molecular biology.
Traditional frontal lesson with ppt slides
Scientific articles and reviews Reference book: Francesco Clementi, Guido Fumagalli, General and Molecular Pharmacology Principles of Drug Action. John Wiley & Sons Inc, 2015. ISBN 9781118768570
Assessment methods and Criteria
The exam consists of an oral test in which three topics are required to be covered. The duration of the oral test is 30 minutes.