Division of Gene Therapy & Regenerative Medicine (GTRM)
Convincing evidence continues to emerge from clinical trials that gene therapy is yielding therapeutic effects in patients suffering from a wide range of diseases. Moreover. the use of adult stem cells and the reprogramming of cells towards pluripotency are being heralded as some of the most promising developments in regenerative medicine at the forefront of medical innovation. This mirrors worldwide efforts in some of the leading research institutes and universities to consolidate gene therapy. stem cells and/or regenerative medicine as some of their prime research activities (e.g. Harvard University. Stanford University. University College London. San Rafaelle Institute-Milano. German Cancer Research Center-Heidelberg).
We therefore believe that it is important that VUB does not lag behind and plays a leadtng and active role in these emerging fields. The mission of the Division of Gene Therapy and Regenerative Medicine (GTRM) is therefore to consolidate a competitive inter-disciplinary program in molecular medicine that focuses primarily on gene therapy and regenerative medicine. The GTRM research program builts upon the nearly 20 years of research of this group in these fields and benefits from established international networks.
The GTRM research prog@m is at the nexus of applied/translational research. hypothesis-driven fundamental research and technology development all of which are inter-related:
(I) Applied translational research:
- to develop and validate gene and cell therapy for major health-and life-threatening diseases with specific emphasis for monogenetic diseases and certain forms of cancer.
- to understand the molecular. cellular and immune mechanisms that influence the outcome of different gene/cell therapy approaches.
- to conduct translational gene therapy studies in large animal models in anticipation of moving forward to the clinic and initiate clinical trials in affected patients.
(II) Hypothesls-drlven fundamental research:
- to unravel the molecular mechanisms and pathways underlying various (patho)physiologic processes important in human health and disease. taking advantage of our state of the art viral and non-viral vector-based gene transfer technologies.
- to generate disease-specific induced pluripotent stem cells OPS) to better understand disease mechanisms and stem cell (coaxed} differentiation and to develop new therapeutic modalities
(III) Technology development:
- to consolidate a broad state-of-the-art technology platform for gene delivery and gene therapy based on the latest viral and non-viral vector systems.
- to establish a state-of-the-art technology platform for regenerative medicine and stem cell biology based on induced pluripotent stem cells (iPS)
This GTRM research program goes beyond gene and cell therapy sensu stricto and addresses broader issues of medical signifiance. taking advantage of the available expertise. This modus operandi has been particulary useful to establish collaborative networks and to increase our scientific output in terms of scientific publications. patent applications. PhDs • grant and industrial partnerships and contracts. In particular. the availibility of a state of the art platforms of gene delivery vectors and induced pluripotent stem cells (iPS) will consolidate existing and foster new collaborations with various groups on the VUB Medical Campus in Jette and the VUB Science Campus in Etterbeek.
Though we have a long-standing interest in liver-borne diseases (e.g. hereditary disorders: hemophilia. complex diseases: hepatocellular carcinoma). the GTRM research program clearly goes beyond liver pathologies per se and therefore transcends the research activitites of CYTO/LIVR which focuses almost exclusively on liver fibrosis. The intrinsic broad and multidiciplinary aspect of the GTRM research program is consistent with the broad range of publications that the group recently published.