System Pharmacology and Cellular and Gene Therapy

Discipline Integrated course
Denomination System Pharmacology and Cellular and Gene Therapy
Modules 2
CFU 10



This teaching course includes the following modules
Module System Pharmacology and pharmacogenetics
Credit 5
SDS BIO/14
Description Learning objectives:
At the end of the course, students are expected to acquire knowledge and competence regarding the main classes of drugs for the treatment of human diseases
In particular, major focus will be on the molecular targets of drug action, and on the genetic basis of the interindividual and interethnic variability in drug response.
Contents:
1
Historical background, significance and clinical relevance of pharmacogenetis and pharmacogenomics for drug efficacy and toxicity prediction
2
Pharmacology and pharmacogenetics of drugs acting on the gastrointestinal system: agents for the control of gastric acidity and peptic ulcers, laxatives, antidiarrheal drugs, antiemetics and drugs against motion sickness
3
Pharmacology and pharmacogenetics of drugs acting on the endocrine system: oestrogens, progestins, hormonal contraceptives, corticosteroids.
4
Pharmacology and pharmacogenetics of drugs acting on the central nervous system: antipsychotics, antidepressants, hypnotics and sedatives, anticonvulsants, opioid analgesics, drugs for the treatment of neurodegenerative diseases (anti-Parkinson, anti-Alzheimer)
Pharmacogenomics of the blood-brain barrier and drug resistance.
5
Pharmacology and pharmacogenetics of drug addiction (cocaine, MDMA, other psychostimulants).
6
Pharmacology and pharmacogenetics of drugs acting on the cardiovascular system: blood pressure lowering agents, diuretics, drugs against heart failure
Genetic basis of drug-induced arrhythmias
7
Pharmacology and pharmacogenetics of lipid-lowering drugs.
8
Pharmacology and pharmacogenetics of antiasthmatics
9
Pharmacology and pharmacogenetics of antidiabetic drugs: insulin, oral hypoglycemics
10
Pharmacology and pharmacogenetics of anti-platelet drugs and anticoagulants.
11
Pharmacology and pharmacogenetics of antineoplastics.
Module Cellular and Gene Therapy
Credit 5
SDS BIO/12
Description Learning objectives:
The aim of the course is to provide students with knowledge and elements of critical discussion on the methodological approaches for gene and cell therapy as well as applications of these methods in both animal models and humans.
Contents:
1) Overview of gene therapy - Definition of vectors and target cells; types of transgenes and applications; regulation of the expression; primary cell cultures; cell banking, and applications of flow cytometry and cell sorting; regulatory aspects of the production of drugs for gene and cell therapy.
2) non-viral vectors – naked plasmids; physical and chemical methods for DNA transfer and their applications; RNA interference and its main applications for therapeutic purposes.
3) adenoviral vectors - Types of adenoviral vectors; toxicity; applications in vaccine and gene therapy.
4) AAV Vectors – vector development; identification of serotypes for alternative tropism; main applications in therapy.
5) Retroviral and lentiviral vectors - Features carriers and differences; pseudotyping; methods of production also for clinical use.
6) stem cells - Definition; Embryonic stem cells; generation, use and applications in gene therapy; Stem cells from adult tissues; Stem cells from bone marrow and use in regenerative medicine; iPS cells and their applications.
7) Tissue regeneration - Identification and use of cells with osteoblastic potential and strategies to induce osteoblastic differentiation; regeneration of epidermis and cornea; identification and culture of stem cells for specific applications; biomaterials and their use in regenerative medicine.
8) Gene therapy of inherited diseases - Development and applications of gene therapy strategy for the treatment of metabolic diseases in humans; Gene therapy for congenital immunodeficiencies and applications in humans; genotoxicity and potential solutions.
9) Gene and regenerative therapy for common diseases - therapy of cardiovascular diseases and regeneration of cardiac tissue; development of medical devices for gene transfer; regeneration of myocardial tissue by reprogramming; therapeutic angiogenesis; gene and cell therapy of diabetes mellitus.
10) Gene therapy from bench to bedside: study of the development of gene therapy drugs from the evaluation of the metabolic disease to the marketing of the drug for gene therapy.
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