Projects
Analysis of JAK-inhibitor treatment for STAT1 gain-of-function and development of a CRISPR-based gene therapy approach KU Leuven
Primary immunodeficiencies (PIDs) are rare inherited disorders affecting the immune system presenting with increased susceptibility to pathogens and often associated with severe non-infectious comorbidities. The poor understanding of PIDs makes them difficult to diagnose and thus, challenging to treat or cure. The monogenic nature of several PIDs converts them in the perfect target for precision medicine. In this project, I focus in autosomal ...
Development of surgical assistive technology for intraocular microsurgery KU Leuven
Our eyesight is by far our most developed sense, playing a vital role in nearly all aspects of everyday life. As such, severe vision loss can have a devastating impact on one’s life. Advancements in medicine have led to effective treatment methods for a substantial range of eye diseases. Nevertheless, several retinal disorders remain a common cause of vision loss. At the time of writing, an estimated 244 million people worldwide suffer from a ...
Characterization of growth regulatory protein complexes in plants via multi-functional TAP technology. Ghent University
Proteins are one of the key components in the complex regulatory networks that underlie biological processes. Although some work independently, most proteins form interactions with other proteins or macromolecules, such as RNA and DNA, to perform their function. To aid functional annotation in plants in a system-wide manner, a wide variety of techniques have been constructed over the years
(Chapter 1), which often imply a ...
CO-cultivation platform technology Research Institute for Agriculture, Fisheries and Food
Despite its economic and environmental benefits, the flow from Ri technology to commercial plant breeding is still limited due to a number of bottlenecks identified in each of the three major steps of the technology: 1) Induction of hairy roots is strongly genotype-dependent and morphological identification is often not possible; 2) Regeneration efficiency of shoots from hairy roots is ...
PAVING the way: Patient preferences to Assess Value IN Gene therapies KU Leuven
Drug development is increasingly focusing on precision medicine, Advanced Therapy Medicinal Products (ATMPs) including gene therapies, and orphan drugs. With the rise of gene therapies, that may have the potential to create permanent effects in patients, decision-making (on the level of marketing authorization, health technology assessment (HTA) and payer decision making, and shared-decision making between patients and physicians) will ...
Gene therapy for cystic fibrosis in a mouse model using adeno-associated viral vectors KU Leuven
Cystic fibrosis (CF) is the most common monogenic life-threatening disease in the Caucasian population, caused by mutations in CFTR, a chloride/bicarbonate channel that regulates fluid transport across epithelium of different organs (airways, pancreas, intestine, sweat glands and vas deferens). CF affects multiple organs, but lung pathology is the major clinical manifestation. Mutations in the CFTR gene lead to an imbalance ...
Designing cell-type specific enhancers for CNS Gene Therapies. University of Antwerp
Enhancer-AI: AI-driven modelling and design of cell type specific enhancers for gene therapy KU Leuven
1) Summary of scientific goals
State-of-the-art gene therapies hold great potential to cure diseases with a high unmet medical need. However, despite major breakthroughs in gene therapy research, clinical implementation is hampered by (1) safety issues due to high vector dosing, (2) inefficient and off-target gene expression due to suboptimal transgene expression control and vector packaging limitations, and (3) high production costs. In ...