- Docente: Carla De Giovanni
- Credits: 5
- SSD: MED/04
- Language: Italian
- Teaching Mode: Traditional lectures
- Campus: Bologna
- Corso: Second cycle degree programme (LM) in Medical Biotechnology (cod. 8859)
Learning outcomes
At the end of the course students will:
- know basic concepts of gene therapy, technologies of gene transfer, therapeutic strategies, efficiency and safety issues;
- know inherited and acquired diseases that could benefit from gene therapy;
- know principles of regulatory issues for gene therapy;
- be able how to identify potential targets and approaches for gene therapy; be able to critically evaluate features, innovation, safety of reports on gene therapy studies;
- know how to present a gene therapy approach, from the description of pathology to conventional and biotechnological strategies;
- know how being up to date on laws, methods, techniques and instruments
Course contents
INTRODUCTION
- Fundamentals, nomenclature, chronology
- Sources: journals, scientific societies, Internet website
MOLECULAR BASES OF GENE THERAPY
- Fundamentals of gene transfer
- Viral vectors for gene therapy
- Non-viral gene transfer: Plasmids and DNA vaccines; Balistic methods; Liposomes; Engineered zinc-finger nucleases
- Targeting: Transductional targeting; Transcriptional targeting; Inducible and tissue-specific promoters
- Ablative strategies: Transduction of antisense constructs; Antisense oligonucleotides; Ribozymes; Intracellular antibodies; RNA interference; engineered nucleases, CRISPR approaches and gene editing
CANCER GENE THERAPY
- Specific aspects of cancer gene therapy: Normal and tumoral cell recipients; Proliferation-dependent vectors; Transduction efficiency and bystander effects
- Gene therapy of the transformed phenotype: Oncosuppressor gene transfer; Oncogene inhibition; Genes overexpressed in tumor cells; Gene therapy targeting cancer neoangiogenesis
- Immunological cancer gene therapy: Fundamental tumor immunology and immunotherapy; Cytokine genes; Antigen genes (Major Histocompatibility Complex, Co-stimulator genes; Tumor antigens
- Changing drug response: Drug sensitivity genes; Drug resistance genes
- Combined therapy
GENE THERAPY OF INHERITED OR ACQUIRED DISEASES
- Monogenic inherited diseases: General aspects; Specific diseases (Primary immunodefiiciencies; Cystic fibrosis; Inherited coagulopathies)
- Gene therapy of infectious diseases: HIV infection
- Gene therapy of ischemic diseases
- Gene therapy of ocular diseases
SAFETY, LAWS, BIOETHIC CONCERNS
- Safety issues in preclinical and clinical gene therapy
- Italian and international laws and safety
- Ethic issues concerning fetal or germinal cell gene therapy
Readings/Bibliography
Textbook 'Terapia genica' (Pierluigi Lollini, Carla De Giovanni e Patrizia Nanni) and powerpoint presentations available at campus.cib.unibo.it
Supplementary suggested text: Giacca, Terapia genica, Springer 2011
Teaching methods
Lectures
Interactive summaries
Assessment methods
On-going: Written tests will be administered in itinere (multiple choice and case study), along with student assignments in workgroups. Marks are explained in the downloadable Course outline
Oral exam.
Both on-going and oral exams will evaluate the achievement of the Learning objectives:
- Define the main technologies of gene transfer and compare them in terms of therapeutic approaches, efficiency and safety
- Explain inherited and acquired diseases that could benefit from gene therapy
- Know principles of gene therapy regulation
- Plan a gene therapy approach, from the description of pathology to conventional and biotechnological strategies
- Choose the gene therapy approach that fits each disease
- Critically evaluate features, innovation, safety of reports on gene therapy studies
- Be able to describe in detail a gene therapy approach, enlightening its advantages and disadvantages
- Be able to keep up-to-date on rules, methods, technologies and devices, through scientific literature, gene therapy websites and data banks
On-going tests and/or oral exam will contribute to the final mark of the Integrated Course 90337 TERAPIE MEDICHE INNOVATIVE, with proportion to the number o credits assigned to Gene Therapy
Teaching tools
PC and videoprojector
Didactic material showed in lectures can be downloaded by students from e-learning website (login with student credentials). Students are invited to attend lectures bringing a printed copy of didactic materials.
Office hours
See the website of Carla De Giovanni
SDGs
This teaching activity contributes to the achievement of the Sustainable Development Goals of the UN 2030 Agenda.