Coordination: Miguel Prazeres and Ana Coroadinha

 

 

Objectives

 

This course aims to teach the fundamental concepts, tools and applications of gene therapy mainly in the context of regenerative medicine.

 

Program

 

1. Gene therapy: therapeutic (e.g. monogenic diseases, immunotherapy of cancer, tissue repairing) and prophylactic (e.g. infectious diseases). 2. Viral vectors: integrative and non-integrative viruses. 3. Non-viral vectors: naked and complexed (liposomes, nano- and microparticles) plasmids. 4. Routes of administration and delivery methodologies. 5. Gene therapy with autologous genes (substitution, blockage, increased expression) and heterologous genes (immune stimulation, cell suicide, cell labelling). 6. Strategies to escape gene delivery barriers: plasmid shielding, increased nuclease resistance and increased transcription levels. 7. Ethical issues in gene therapy.

 

Evaluation Methodology

 

70% - Presentation of a seminar comprising the critical analysis of a scientific article published in specialized journals.
30% - Writing of an Abstract Essay.