Publicaties
Improved animal models for testing gene therapy for atherosclerosis Universiteit Antwerpen
Gene therapy delivered to the blood vessel wall could augment current therapies for atherosclerosis, including systemic drug therapy and stenting. However, identification of clinically useful vectors and effective therapeutic transgenes remains at the preclinical stage. Identification of effective vectors and transgenes would be accelerated by availability of animal models that allow practical and expeditious testing of vessel-wall-directed gene ...
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
Hemophilia is an inherited bleeding disorder caused by a deficiency of functional clotting factors VIII or IX in the blood plasma. The drawbacks of the classical protein substitution therapy fueled interest in alternative treatments by gene therapy. Hemophilia has been recognized as an ideal target disease for gene therapy because a relatively modest increase in clotting factor levels can result in a significant therapeutic benefit. ...
Emerging potential of transposons for gene therapy and generation of induced pluripotent stem cells. Vrije Universiteit Brussel
Effective gene therapy requires robust delivery of the desired genes into the relevant target cells, long-term gene expression, and minimal risks of secondary effects. The development of efficient and safe nonviral vectors would greatly facilitate clinical gene therapy studies. However, nonviral gene transfer approaches typically result in only limited stable gene transfer efficiencies in most primary cells. The use of nonviral gene delivery ...
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 ...
Gene therapy strategies for hemophilia: benefits versus risks KU Leuven
Hemophilia is an inherited bleeding disorder caused by a deficiency of functional clotting factors VIII or IX in the blood plasma. The drawbacks of the classical protein substitution therapy fueled interest in alternative treatments by gene therapy. Hemophilia has been recognized as an ideal target disease for gene therapy because a relatively modest increase in clotting factor levels can result in a significant therapeutic benefit. ...
Recent developments in transposon-mediated gene therapy. Vrije Universiteit Brussel
INTRODUCTION: The continuous improvement of gene transfer technologies has broad implications for stem cell biology, gene discovery, and gene therapy. Although viral vectors are efficient gene delivery vehicles, their safety, immunogenicity and manufacturing challenges hamper clinical progress. In contrast, non-viral gene delivery systems are less immunogenic and easier to manufacture. AREAS COVERED: In this review, we explore the emerging ...