Vrije Universiteit Brussel (VUB)
The Free University of Brussels (VUB) has over 15,000 students and 6,500 staff members, making it one of the largest employers in the Brussels Capital Region. The University Medical Centre (UMC) is composed of the Brussels University Hospital and the VUB Faculty of Medicine and Pharmacy. The research is organized in 10 thematic “Clusters of Excellence” of which the Department of Gene Therapy & Regenerative Medicine (GTRM) is an integral part. Biomedical research at VUB focuses on understanding the molecular basis of and developing new treatments for diseases of major medical significance with frequent contributions in top-tier journals.
Within the MYOCURE project, Prof. Dr. Marinee Chuah, Deputy Director of the Department of Gene Therapy & Regenerative Medicine at VUB, will coordinate the scientific parts of the project and will lead the VUB research team. The core competencies of VUB-GTRM are: (i) development and validation of a new bio-informatics platform for rational vector design; (ii) pre-clinical development of safe and effective gene therapy approaches for hereditary monogenetic diseases, including muscle disorders; (iii) expertise in targeted tissue-specific gene transfer (i.e. heart, muscle, liver); (iv) insights into the immune consequences of gene therapy and immuno-modulatory approaches; (v) regulatory and translational gene therapy expertise and (vi) experience in pre-clinical mice, dog and Non-Human Primate (NHP) models for gene therapy.
All these competencies of VUB-GTRM constitute the foundation of MYOCURE and contribute significantly towards the specific objectives of the project, aimed at developing an innovative and validated gene therapy platform to cure rare hereditary muscle disorders.