Objectives of Programme's Learning Outcomes

• Scientific skills
• Use acquisition and data analysis software specific to biomedical sciences
• Be a responsible researcher: - Know how to base their reasoning on data obtained from scientific literature. - Know how to integrate an ethical dimension in their reasoning. - Be loyal (to the facts, to the team, and to intellectual property). - Do not tamper with results. - Do not exploit the work of others. - Demonstrate experimental rigor.
• Scientific skills
• Use acquisition and data analysis software specific to biomedical sciences
• Be a responsible researcher: - Know how to base their reasoning on data obtained from scientific literature. - Know how to integrate an ethical dimension in their reasoning. - Be loyal (to the facts, to the team, and to intellectual property). - Do not tamper with results. - Do not exploit the work of others. - Demonstrate experimental rigor.

Learning Outcomes of UE

The general strategy of gene therapy involves the replacement of a mutated gene that causes a disease by a functional copy in the patient. The course presents the initial concept and its evolution for the past 15 years including the development of antisense strategies (RNA interference, ribozymes, oligonucleotides...).
At the end of this course, the students will be familiar with the basic concepts in gene therapy, the analysis of gene/function in given candidate pathologies for such a treatment, and the experimental and clinical strategies involved. They will be able to understand and criticize recent scientific publications related to this field.
 

Content of UE

Impact of a mutation on the phenotype. Loss and gain of function, recessive and dominant phénotypes, dominant negative, haploinsuffisancy. Genic therapy: ex-vivo and in vivo stratégies, target cells, stem cells. Episomal or integrative therapeutic gene in target cells. Viral and non-viral vectors: advantages and disadvantages. Antisesne stratégies, siRNA, miRNA, antisense oligonucleotides, ribozymes, aptamers, RNA decoys, trans splicing, mutation corrections, zinc-finger and TALE nucléases, CRISPR/Cas9. Examples of genic therapies on different pathologies and succesful.
 

Prior Experience

Molecular Biology (including compléments or regulation of genic expression )

Type of Assessment for UE in Q1

• Presentation and/or works
• Written examination

Q1 UE Assessment Comments

50/50 weighting for written exam and presentation

Type of Assessment for UE in Q3

• Written examination

Q3 UE Assessment Comments

single test

Type of Resit Assessment for UE in Q1 (BAB1)

• N/A

Q1 UE Resit Assessment Comments (BAB1)

N/A