Three European MSCA-DN grants for research at Radboudumc European funding for research on cellular protrusions and improving antisense therapy

Radboud university medical center is the coordinator of two large projects funded by the European Commission, involving many research institutions and companies from various countries. Radboudumc is also a partner in a third project. The researchers are developing therapies using so-called antisense oligonucleotides and investigating the role of cellular protrusions in health and disease.

Expanding the therapeutic use of oligonucleotides

Project coordinator: Alex Garanto (Radboudumc) 
Project Name: A European Training Program to Foster the Full Therapeutic Potential of Antisense Technology across Tissues (EFFeCT)

RNA molecules are increasingly gaining attention as therapeutic strategy for medical care, such as RNA vaccines. A different type of RNA molecules, called antisense oligonucleotides, hold promise as therapeutics for various common and rare conditions. These oligonucleotides can lead to changes in protein production, by for example increasing the levels of the correct protein or reducing the amount of harmful proteins. Despite the potential of these molecules, in the past 25 years, only 21  molecules have been approved by the FDA or EMA. This project aims to overcome certain obstacles that still hinder the translation to patients. EFFeCT researchers will solve delivery issues in specific tissues, increase knowledge about oligonucleotides, and create a roadmap for these drugs in Europe. This will significantly boost the field of oligonucleotide research.

The role of the cellular antenna in health and disease 

Project coordinator: Ronald Roepman (Radboudumc) 
Project Name: European Training Program for Deconvolution of Multi-scale Cilia Function in Health and Disease by Integrating Machine Learning-AI Approaches (Cilia-AI)

Cilia are small, elongated projections present on nearly all cells in the body. They play a crucial role in the development and maintenance of cells, tissues, and organs, acting like sensory antennas. Cilia are essential for functions such as hearing, smell, respiration, excretion, and reproduction. Malfunctioning cilia can lead to more than 35 severe inherited diseases, known as ciliopathies, affecting up to one in four hundred people. In this project, researchers will unravel how cilia regulate bodily functions at various levels, from molecules to organs, and how disruptions lead to disease. They will use a multidisciplinary approach, including multi-omics and advanced microscopy, and will develop artificial intelligence (AI) to analyze the large amounts of data generated by this approach.

Oligonucleotide therapy for myotonic dystrophy

Coordinators of Radboudumc as a participating center: Rick Wansink en Hans van Bokhoven 
Project Name: Interdisciplinary doctoral training on oligonucleotide-based therapies for myotonic dystrophy (ENTRY-DM)

Myotonic dystrophy is the most common inherited muscular dystrophy in adults, affecting 60,000-70,000 people in Europe. It is a complex disorder with significant variation in genetic background and clinical symptoms, making it difficult to establish clear links between genetics and symptoms. This complexity also makes it challenging to develop a therapy that can ultimately be used to treat the various symptoms of myotonic dystrophy. Antisense oligonucleotides show promise as a therapeutic. In this project, researchers aim to improve scientific and technological knowledge about myotonic dystrophy. They will use a multidisciplinary approach, including computational models, to study disease mechanisms, optimize oligonucleotide therapy, and pave the way for future clinical trials.

About the grant

The three projects have been awarded under the MSCA Doctoral Networks 2023 call by the European Commission. The MSCA Doctoral Networks aim to train creative, entrepreneurial, innovative, and resilient doctoral candidates capable of addressing current and future challenges and turning knowledge and ideas into products and services that bring economic and social benefits.

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