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Blood highlights groundbreaking research at the VUB

"A David promoter with Goliath strength"
A commentary in Blood highlights groundbreaking research at the VUB.

Prof . Dr Thierry VandenDriessche and Prof. Dr Marinee Chuah of the Department of Gene Therapy and Regenerative Medicine (GTRM) (Faculty of Medicine & Pharmacy) received comments on their paper, which was published in the reputed scientific journal 'Blood' on May 15. For the past 20 years, the research team has been conducting translational and basic research in gene therapy for the treatment of severe genetic diseases, such as haemophilia. In gene therapy, viruses are used as a transport vehicles (designated as vectors) in order to bring the new therapeutic DNA into the cells and cure the underlying disease. To achieve this, a promoter is needed as a "molecular engine" to drive expression of the therapeutic gene.

The past years, the GTRM research team identified a small DNA fragment that enhances the effectiveness of this promoter at least 10 times , that then could serve as a sort of 'molecular turbo charger' to boost the overall performance of the gene therapy vector. This discovery has important implications for gene therapy. Indeed, the expression of the therapeutic gene is restricted to the desired target tissue and not in other organs, minimizing the risk of potential side-effects while improving the effectiveness of the gene therapy vector at lower doses. This breakthrough could facilitate the treatment of patients by gene therapy, potentially reducing costs of current therapies and obviating the need for repeated treatment. The paper in ‘Blood’ specifically relates to liver disease. However, this emerging new concept -at the nexus of gene therapy and computational genome-wide DNA analysis- has broad implications for other diseases, including cardiac, muscular and metabolic disorders. 

This research was funded in part by the Industrial Research Fund (IOF) (Groups of Expertise in Applied Research & Proof of Concept Grants) & Strategic Research Program (Grower Grant), FWO, EU Framework Program 7, AFM.

VUB-prof Thierry VandenDriessche is head of the Department of Gene Therapy and Regenerative Medicine of VUB and has been elected a member of the Board of Directors of the American Society of Gene & Cell Therapy.

The American Society of Gene Therapy (ASGT) is a prestigious medical and scientific association specialized in the development and application of genetic cell therapy. The association also aims to raise awareness and public education regarding gene therapy.