5 ECTS credits
140 u studietijd
Aanbieding 1 met studiegidsnummer 4018230FNR voor alle studenten in het 1e semester met een gespecialiseerd master niveau.
"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, through scientific papers that are available online and constitute the basis of the exam).
De beoordeling bestaat uit volgende opdrachtcategorieën:
Examen Mondeling bepaalt 100% van het eindcijfer
Binnen de categorie Examen Mondeling dient men volgende opdrachten af te werken:
The exam entails a 12 min presentation of an article of your choice based on the papers that are available on the Canvas platform in the Gene Therapy and Gene Editing module. Typically, the talks should be based on maximum 15 slides. The talk is followed by an interactive session of about 5 min, where you will be asked questions on (i) the content of the presentation and (ii) its relationship with the course (for example: if you choose a topic related to lentiviral vectors, you would need to know the properties of these vectors and how they are produced and justify why this particular vector was used in this study and not another one, like AAV). The Powerpoint slides of the Gene Therapy lectures are available on Canvas.
The exam/presentation will take place outside the regular exam period.
The presentation has to be made using Powerpoint, keynote or a similar format and should encompass an introduction, objectives, results, conclusion. It should also include a critical assessment of the paper (i.e. what are the limitations of the study and how can we improve it?).
The objective is really to assess whether you can apply the concepts that you have learned in the lectures in a new context based on some of the top recent papers in the field in high impact factor journals (Nature journals, Science, New Engl J Med, etc. ...). All students from that group stay present during the entire session to ask questions in an interactive manner.
Your final mark will depend on:
1) Quality and structure of the slides
2) Clarity of the oral presentation
3) Answers to questions (Understanding of the course material)
4) Critical assessment of the paper's findings
Deze aanbieding maakt deel uit van de volgende studieplannen:
Master in de biomedische wetenschappen: biomedisch onderzoek
Master in de biomedische wetenschappen: management en bedrijfsleven
Master in de biomedische wetenschappen: klinische biomedische wetenschappen
Master in de biologie: moleculaire en cellulaire biowetenschappen
Master of Biology: Molecular and Cellular Life sciences (enkel aangeboden in het Engels)
Master of Biomedical Research: Standaard traject (enkel aangeboden in het Engels)
Educatieve master in de gezondheidswetenschappen: standaard traject (120 ECTS, Etterbeek)