 | Study programme | Français | |
 | Complements of molecular biology and genic therapy | ||
Activité d'apprentissage à la Faculty of Medicine and Pharmacy |
| Code | Lecturer(s) | Associate Lecturer(s) | Subsitute Lecturer(s) et other(s) |
|---|---|---|---|
| M-BIMO-003 |
|
| Language of instruction | Language of assessment | HT(*) | HTPE(*) | HTPS(*) | HR(*) | HD(*) | Term |
|---|---|---|---|---|---|---|---|
| Français | Français | 15 | 15 | 0 | 0 | 0 | Q1 |
Content of Learning Activity
Impact of a mutation on the phenotype. Loss and gain of function, recessive and dominant phenotypes, dominant negative, haploinsufficiency. Gene therapy: ex-vivo and in vivo strategies, target cells, stem cells. Episomal or integrative therapeutic genes in target cells. Viral and non-viral vectors: advantages and disadvantages. Antisense strategies, siRNA, miRNA, antisense oligonucleotides, ribozymes, aptamers, RNA decoys, trans splicing, mutation corrections, zinc-finger and TALE nucleases, CRISPR/Cas9. Examples of gene therapies on different pathologies and their successes.
Required Learning Resources/Tools
Not applicable
Recommended Learning Resources/Tools
informations complémentaires sur la plateforme Moodle
Other Recommended Reading
Not applicable
Mode of delivery
- Face to face
Type of Teaching Activity/Activities
- Cours magistraux
Evaluations
The assessment methods of the Learning Activity (AA) are specified in the course description of the corresponding Educational Component (UE)