Viral Vectors for Gene Therapy: repurposing viruses to study and cure genetic disorders

Presented by Dr Rik Gijsbers, Associate Professor, Laboratory of Viral Vector Technology and Gene Therapy, Department of Pharmaceutical and Pharmacological Sciences, Faculty of Medicine, KU Leuven

Over the past three decades gene therapy has evolved to a clinical reality with several gene therapeutic products reaching the market in the past few years, providing treatment for a small subset of otherwise untreatable inherited and acquired genetic disorders.

Gene therapy aims to achieve durable expression of the therapeutic genetic information at a level sufficient to alleviate or cure disease symptoms with minimal adverse events. Virus-derived vectors (viral vectors) have evolved from laboratory tools to gene transfer agents with two basic strategies: (i) an integrating retroviral vector (RV, LV) is introduced into a stem cell (ex vivo) allowing the genetic information to be passed on to every daughter cell or (ii) a non-integrating adeno-associated viral vector (rAAV) is provided to a long-lived, differentiated or slowly dividing cells (in vivo), with the transferred DNA residing extrachromosomally.

More than 3000 trials approved worldwide have provided essential insights and knowledge regarding gene therapy in general, but also learned that each new disorder requires to be approached in its own unique way. Even though gene therapy theoretically caries the potential to provide a cure, this may not necessarily be a life-long one.