About Gene therapy: main approaches and biosafety issues​

Gene therapy: main approaches and biosafety issues​

Instructors: Gijsbert van Willigen, Suzanne Loret

12 November 10:00 - 15:30 CET (lunch break 11:30 - 14:00)
13 November 10:00-12:00 CET

Though the idea for gene therapy was put forward in 1972, it was not until 2003 in China and 10 years later in US, that DNA drug actually appeared on the market. Since then, at least nine gene therapies have been approved for a few cancer types, genetic disorders and viral infections and a great number of research projects and clinical trials are underway in many countries.

Target audience:

  • Laboratory professionals whose activities are involved in molecular and cell biology, including DNA recombinant technologies and cell transfection approaches
  • Health, Safety and Environment (HSE) Professionals involved in the risk assessment of molecular and cell biology activities and gene therapy clinical trials
  • Any person interested by the subject

Learning outcomes:

At the end of the course, participants should be able to:

  • get an accurate understanding of:
    • gene therapy
    • gene therapy main approaches
    • gene vector
  • perform a risk assessment of gene therapy conditions (this capacity will be tested through a group work on study cases)


  • Basics Biosafety: pathogenic organism / genetically modified organism - risk group - contained use - containment level
  • General biology: basic knowledge in cell biology and virology
  • Molecular biology: basic knowledge about cloning - expression - transfection - transduction

The training will focus on:

  • the main approaches in gene therapy (using various gene vectors), with an attention paid on the biosafety aspects in research and development laboratories, as well as on the risk for the patient
  • the viral vectors used frequently in current gene therapy project, with the aim to point out their respective benefits in term of efficacy and safety

Participants will have the opportunity to perform a team-work risk assessment on case studies corresponding to current clinical trials.

CWA 15793 Reference

  • 7.2.1 General principles of microbiology, biochemistry and cell biology
  • 7.2.2 General principles of molecular biology and genetic engineering
  • 7.2.3 Gene therapy activities