90337 - Gene therapy

Course Unit Page

Academic Year 2017/2018

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 AMS campus website http://campus.unibo.it/ (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