| 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 |
Contents
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
Term 1 Assessment - type
- Presentation and works
Term 1 Assessment - comments
Not applicable
Term 2 Assessment - type
- N/A
Term 2 Assessment - comments
Not applicable
Term 3 Assessment - type
- Presentation and works
Term 3 Assessment - comments
Not applicable
Resit Assessment - Term 1 (B1BA1) - Comments
Not applicable
Type of Teaching Activity/Activities
- Cours (cours magistraux; conférences)
- Exercices dirigés / utilisation de logiciels / démonstrations