Objectives of Programme's Learning Outcomes

• Scientific skills
• 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.
• Professional integration skills
• Be aware of ethical issues and apply ethical codes specific to life sciences in their behaviour
• Demonstrate their ability to research, analyse and synthesise: - Research, analyse, use different sources of information and materials related to biomedical sciences, format them to draw up a summarised document, produced and broadcast them on digital media. - Carry out a study, identify and raise an issue in a predefined context, build and develop an argument, interpret the data and results, develop an overview, and propose extensions. - Question themselves, think critically, debate, and defend their ideas.
• Make a presentation illustrating arguments adapted to the object, the circumstances and the public: speak in public

Learning Outcomes of UE

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

Prior Experience

molecular biology and regulation of genic expression

Type of Assessment for UE in Q1

• Presentation and works

Q1 UE Assessment Comments

Not applicable

Q2 UE Assessment Comments

Not applicable

Type of Assessment for UE in Q3

• Presentation and works

Q3 UE Assessment Comments

Not applicable

Q1 UE Resit Assessment Comments (BAB1)

Not applicable