Projects
Gene editing for cystic fibrosis - tackling the translatability hurdle KU Leuven
This project is focussed on novel gene editing modalities and delivery methods to develop a gene therapy treatment for the monogenetic disease cystic fibrosis which primarily manifests with lung disease. Objectives: (1) To use gene editing (in particular base and prime editing) for efficient correction of CFTR (gene mutations in CFTR are responsible for cystic fibrosis) in primary human organoid and airway models. (2) To develop safe and ...
Human germline gene editing to overcome transmission of infertility: a proof-of-concept study Ghent University
The recent development of the CRISPR/Cas9 technology as a versatile and efficient tool has enabled human germline gene editing and can be applied to correct abnormal DNA sequences during fertilization. However, its future implementation for therapeutic approaches is still heavily debated, not only from an ethical perspective, but also because many uncertainties regarding the safety and efficiency of the technique still exist. More ...
A highly efficient CRISPR/Cas9 gene editing method for difficult to transform arthropods Ghent University
During the ERC consolidator grant POLYADAPT, an efficient gene editing system was discovered which allows to genetically transform the spider mite, Tetranychus urticae, a main crop pest. This species belongs to the chelicerates, the second largest group of arthropods including mites, ticks and spiders, that are notoriously difficult to transform. Our invention not only accomplished for the first time gene knock-out in mites, but also allows ...
Development and functional validation of a gene editing approach in hematopoietic precursor cells for Wiskott-Aldrich Syndrome Ghent University
The Wiskott-Aldrich syndrome (WAS) is an X linked disease caused by a defect in the WAS gene.
Affected individuals suffer from recurrent infections, eczema, bleeding, auto-immune symptoms
and an increased risk of leukemia. Since WAS protein is involved in the signal transfer from the T
cell receptor to the cytoplasm, the lack of this protein will result in a suboptimal immune
responses to pathogens. In addition, T cell ...
Targeted editing of the PSIP1 gene encoding LEDGF/p75 protects cells against HIV infection KU Leuven
Gene therapy has long held promise to correct a variety of human diseases. Discovery of the Clustered Regularly-Interspaced Short Palindromic Repeats (CRISPR), the mechanism of the CRISPR-based prokaryotic adaptive immune system (CRISPR-associated system, Cas) and its repurposing into a potent gene editing tool has revolutionized the field of molecular biology and generated excitement for new and improved gene therapies. Additionally, the ...
CMOS-based multielectrode array for spatially controlled single-cell gene editing and monitoring KU Leuven
I will exploit a novel CMOS microelectrode array chip (MEA) to monitor and genetically modify cells at the single-cell level. First, I will use impedance measurements and cell imaging to identify cell types with distinct functional properties (e.g. proliferation rate, impedance signatures) from heterogeneous glioblastoma models. Second, I will locally electroporate cells to fluorescently tag them and genetically edit their genome, while ...
A wheat and barley gene editing facility KU Leuven
A gene editing strategy for refractory CFTR mutations causing cystic fibrosis. KU Leuven
The goal of our project is to develop CRISPR-Cas gene editing therapy for CFTR mutations refractory to current CFTR modulators. A functional rescue will be assessed in human CF intestinal organoïds and airway cultures.
A method for efficient CRISPR/Cas9 Gene editing in difficult to transform arthropods Ghent University
In this concepTT, we want to capitalize on a unique gene editing method that we developed for the spider mite Tetranychus urticae. Mites - together with ticks, spiders and scorpions - belong to the Chelicerata, the second largest group of animals on the planet, and are notoriously difficult to genetically transform. We will collect data on variations in the formulation composition that might further improve editing efficiency. We will show ...