5 ECTS credits
140 h study time
Offer 1 with catalog number 4018230FNR for all students in the 1st semester at a (F) Master - specialised level.
"Gene Therapy and Gene Editing"
Basic aspects of Gene Therapy
1. Non-viral gene transfer methods:
- Stable vs. transient transfection (lipofection, calcium phosphate transfection, electroporation, …)
- Transposon-based technology (e.g. Sleeping beauty)
2. Viral gene transfer:
- Retroviral vectors
- Lentiviral vectors
- Adeno-associated viral vectors
- Cell-type specific targeting
- The production methods used to generate these different gene delivery vectors will be outlined in detail. This will also include a more practical approach to vector characterization.
- The properties of each of these gene delivery approaches in terms of efficacy and safety will be outlined in detail. Safety issues will focus on genotoxicity and immune consequences of gene transfer.
3. Concepts in gene transfer:
- ‘Gene editing’ with designer nucleases (ZFNs, CRISPR/Cas9, TALENs): targeted gene correction and integration in ‘safe harbour’ loci: ‘on-target’ & ‘off-target’ effects
- Genetic modification of somatic cells vs. germline cells
- Genetic modification of stem cells (iPS, ES, hematopoietic stem cells): pro’s and con’s for regenerative medicine applications
4. Gene therapy for monogenetic diseases (e.g. hemophilia, muscular dystrophy, Leber congenital amaurosis, hematopoietic disorders, …)
5. Gene therapy for neurodegenerative disorders
6. Gene therapy for cancer
7. Gene therapy for diabetes
Powerpoint slides are accessible through Canvas. The lectures will be conducted alternatingly on the Jette and Etterbeek campus with teleclassing, so that the students who are in Jette do not have to drive to Etterbeek and vice versa. The lectures that are taught in Jette will be transmitted ‘live’ to Etterbeek and vice versa. Discussions between the students and the lecturer and Q&A sessions can readily be implemented with this tele-classing modality.
- Having a fundamental knowledge of the descriptive and functional aspects of the molecular biological basis of gene therapy, gene transfer and gene editing in vitro and in vivo. This implies a level of knowledge and understanding that enables the student to design a biotechnological and biomedical research plan on gene therapy and gene editing.
- To achieve a critical attitude and attitude for scientific argumentation. To be able to practice the studied concepts independently and test these (for example through critical questions during lectures)
The final grade is composed based on the following categories:
Written Exam determines 100% of the final mark.
Within the Written Exam category, the following assignments need to be completed:
This offer is part of the following study plans:
Master of Biomedical Sciences: Management and Business (only offered in Dutch)
Master of Biomedical Sciences: Clinical Biomedical Sciences (only offered in Dutch)
Master of Biology: Molecular and Cellular Life sciences
Master of Biomedical Research: Standaard traject
Master of Teaching in Health Sciences: standaard traject (120 ECTS, Etterbeek) (only offered in Dutch)