Academic Year 2017/2018
- 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
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.
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