Objectives of Programme's Learning Outcomes

• In the field of biological sciences and particularly in the field of the biochemistry, molecular and cell biology, possess highly specialised and integrated knowledge and a wide range of skills adding to those covered in the Bachelor's programme in biological sciences.
• Master communication techniques.
• Communicate, both orally and in writing, their findings, original proposals, knowledge and underlying principles, in a clear, structured and justified manner.
• Master the techniques of written and oral scientific communication in both French and English.
• Develop and integrate a high degree of autonomy.
• Aquire new knowledge independently.
• In the field of biological sciences and particularly in the field of the biochemistry, molecular and cell biology, possess highly specialised and integrated knowledge and a wide range of skills adding to those covered in the Bachelor's programme in biological sciences.
• Master communication techniques.
• Communicate, both orally and in writing, their findings, original proposals, knowledge and underlying principles, in a clear, structured and justified manner.
• Master the techniques of written and oral scientific communication in both French and English.
• Develop and integrate a high degree of autonomy.
• Aquire new knowledge independently.

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 (including complement and/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)

Not applicable