Somatic gene transfer/ Cystic fibrosis
Group leader: Bob J Scholte
Group members: Daniel Klink, Christine d'Oliveira, Rogier Louwen, Pascal van der Wegen.
Somatic gene therapy involves the transfer of a therapeutic gene to the appropriate target cells of a patient. While this basic concept is simple enough, vector systems for clinical application still have to be substantially improved to meet all requirements of safety and efficacy.
Synthetic (non-viral) vectors. In collaboration with research groups in Leiden and Utrecht we aim to create a new generation of synthetic vector systems with improved receptor targeting and intracellular transport capability.
Lentiviral vectors have great potential in gene therapy. In collaboration with Inder Verma (Salk institute) we are generating lentiviral vectors for several inherited disorders including Globinopathies (link naar globin gene regulation), Crigler-Najjar disease (Sophia kinderziekenhuis), Cystic Fibrosis.
Minichromosomes- Stem cell therapy. A novel approach towards somatic gene therapy is the use of minichromosomes, which are able to replicate and segregate during the cell cycle. Another advantage is that these vectors can carry large genes or even gene clusters. Our expertise In the context of an EEC funded program we study transfer of minichromosomes to stem/progenitor cells (Dzierzak), in a model of chronic lung disease.
Murine models of human disease. The vector systems that we develop are being tested in vitro and in vivo using murine models of human disease.
Cystic Fibrosis
Crigler Najjar disease (Sinaasappel SKZ)
Cardiovascular (de Crom)
Globinopaties (Grosveld)
Prostate and Bladder carcinoma (Bangma).