Description

Gene therapy is rapidly emerging to treat rare diseases and includes various strategies such as gene augmentation, antisense oligonucleotides and gene editing. This doctoral school course aims to provide a comprehensive overview of current gene therapy technologies complemented by examples from our university and beyond. Additionally, we will cover the gene therapy development process, from early-stage research and valorisation to human clinical trials.

PROGRAM:

Part 1: Gene therapy - technologies

• 9u-9u35: Overview of genetic therapy strategies (prof. Frauke Coppieters, Ghent Therapy and RNA Group, UGent)

• 9u35-10u10: Gene therapy delivery through nanomedicines (prof. Stefaan De Smedt, Laboratory of General Biochemistry and Physical Pharmacy, UGent)

• 10u10-10u55: Gene therapy delivery through viral vectors (prof. Luk Vandenberghe, Grousbeck Gene Therapy Center, Boston, US)

Break (15min)

Part 2: Gene therapy - applications

• 11u10-11u45: Gene therapy for brain disease with a focus on epilepsy (prof. Robrecht Raedt, 4Brain, UGent)

• 11u45-12u20: Gene therapy for inherited deafness (prof. Frederic Acke, Otology and Audiology group, UGent & UZGent)

• 12u20-12u55: Gene therapy for inherited blindness (prof. Bart Leroy, Dept. of Ophthalmology, UGent & UZGent)

Lunch (13:00-14:00)

Part 3: Gene therapy - development

• 14u-14u35: Valorization and Good Manufacturing Practices (GMP) (dr. Tim Desmet, UGent)

• 14u35-15u10: Gene therapy clinical trials (HIRUZ, UZGent)

• 15u10-15u45: A company’s perspective 

Break (15min)

• 16u-16u35: Science communication (Ineke Imbo, UGent)

• 16u35-17u10: A patient’s perspective (Nederlands)

Questions? Contact Frauke.Coppieters@UGent.be