Improved animal models for testing gene therapy for atherosclerosis Universiteit Antwerpen
Towards a global multidisciplinary consensus framework on haemophilia gene therapy Vrije Universiteit Brussel
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 ...
Gene therapy strategies for hemophilia: benefits versus risks. Vrije Universiteit Brussel
Emerging potential of transposons for gene therapy and generation of induced pluripotent stem cells. Vrije Universiteit Brussel
Hemophilia gene therapy knowledge and perceptions Vrije Universiteit Brussel
Background: Hemophilia gene therapy is a rapidly evolving therapeutic approach in which a number of programs are approaching clinical development completion.
Objective: The aim of this study was to evaluate knowledge and perceptions of a variety of health care practitioners and scientists about gene therapy for hemophilia.
Methods: This survey study was conducted February 1 to 18, 2019. Survey participants were members of the ...
Gene therapy for cardiovascular disease Vrije Universiteit Brussel
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 ...
HEMOPHILIA GENE THERAPY Vrije Universiteit Brussel
Hemophilia A and B are congenital X-linked bleeding disorders caused by mutations in the genes encoding for the blood clotting factor VIII (FVIII) or factor IX (FIX), respectively. Since the beginning of gene therapy, hemophilia has been considered an attractive disease target that served as a trailblazer for the field at large. Different technologies have been explored to efficiently and safely deliver the therapeutic FVIII and FIX genes ...