Publicaties
Predicting genotoxicity of viral vectors for stem cell gene therapy using gene expression-based machine learning KU Leuven
Gene therapy strategies for hemophilia: benefits versus risks. Vrije Universiteit Brussel
Towards a global multidisciplinary consensus framework on haemophilia gene therapy Vrije Universiteit Brussel KU Leuven
INTRODUCTION: With approval of gene therapy for haemophilia likely in the near future, policy frameworks are needed to guide the path forward for this disruptive and novel therapeutic advance.
AIM: The WFH has initiated a series of multi-stakeholder Gene Therapy Round Tables (GTRT) to better understand where guidance is needed and develop initial consensus statements to inform policy.
METHODS: The first day of the 2nd GTRT was ...
Hyperactive PIGGYBAC Transposons for Sustained and Robust Liver-targeted Gene Therapy. Vrije Universiteit Brussel
Muscle Gene Therapy Vrije Universiteit Brussel
Gene therapy for cardiovascular disease Vrije Universiteit Brussel KU Leuven
Gene therapy is a promising modality for the treatment of inherited and acquired cardiovascular diseases. The identification of the molecular pathways involved in the pathophysiology of heart failure and other associated cardiac diseases led to encouraging preclinical gene therapy studies in small and large animal models. However, the initial clinical results yielded only modest or no improvement in clinical endpoints. The presence of ...
Liver-directed lentiviral gene therapy in a dog model of hemophilia B Vrije Universiteit Brussel KU Leuven
We investigated the efficacy of liver-directed gene therapy using lentiviral vectors in a large animal model of hemophilia B and evaluated the risk of insertional mutagenesis in tumor-prone mouse models. We showed that gene therapy using lentiviral vectors targeting the expression of a canine factor IX transgene in hepatocytes was well tolerated and provided a stable long-term production of coagulation factor IX in dogs with hemophilia B. By ...