Project description
European training programme on autosomal dominant retinal diseases
In Europe, around 350 000 individuals are affected by inherited retinal diseases (IRDs), a group of conditions that were once deemed incurable. However, recent advancements have sparked groundbreaking new treatments. The primary hurdles include unresolved genetic diagnoses, unfamiliarity with disease mechanisms, and the development of gene therapy for autosomal dominant IRD (adIRD), which constitutes 25-40 % of all IRD cases. The MSCA-funded ProgRet project will establish a European training network with a specific focus on IRD mechanisms, diagnosis and therapy. The project’s objectives encompass the use of stem cell and animal models to investigate adIRD mechanisms, the enhancement of diagnostics through a single-molecule multi-omics framework, and the creation of innovative treatments based on RNA therapy and CRISPR genome editing.
Objective
ProgRET will create a multidisciplinary and intersectoral European training network focusing on the mechanisms, diagnosis and therapy of dominantly inherited retinal diseases (IRD). IRD represent a major cause of blindness, affecting 350,000 people in Europe. IRD have long been considered incurable, however major advances have led to groundbreaking new treatments. Today, the most important challenges in the IRD field relate to an unsolved genetic diagnosis, unknown disease mechanisms and gene therapy development for autosomal dominant IRD (adIRD), representing 25–40% of all IRD cases. We have demonstrated an emerging role for splicing factors, structural variants and non-coding defects in patients with adIRD, and developed novel disease models and gene therapies for adIRD. ProgRET aims to dissect adIRD mechanisms using retinal stem cell and aquatic animal models, to advance adIRD diagnostics using a single-molecule multi-omics framework, and to develop innovative treatments based on RNA therapy and CRISPR-genome editing. These challenges will be tackled by integrating unique expertise and cutting-edge technology within ProgRET, including (multi-)omics, bioinformatics, functional genomics, RNA biology, gene regulation, stem cell technology, retinal organoids, animal models, genome editing and gene therapy. ProgRET will give Doctoral Candidates (DCs) unparalleled training opportunities in outstanding academic and industrial settings through training-by-research via individual research projects, secondments, and network-wide training sessions. All individual training and research activities will provide each DC with the necessary skills in academic and industrial research. ProgRET will make a career in both sectors attractive and improve their career prospects. Finally, our multidisciplinary network offers a unique opportunity to accelerate the understanding, diagnostics and therapeutics for adIRD in Europe, and to translate research findings to healthcare and society.
Fields of science
- medical and health sciencesmedical biotechnologygenetic engineeringgene therapy
- medical and health sciencesclinical medicineophthalmology
- medical and health sciencesmedical biotechnologycells technologiesstem cells
- natural sciencesbiological sciencesgeneticsRNA
- natural sciencesbiological sciencesgeneticsgenomes
Keywords
Programme(s)
- HORIZON.1.2 - Marie Skłodowska-Curie Actions (MSCA) Main Programme
Funding Scheme
HORIZON-TMA-MSCA-DN - HORIZON TMA MSCA Doctoral NetworksCoordinator
9000 Gent
Belgium
See on map
Participants (7)
6525 GA Nijmegen
See on map
72074 Tuebingen
See on map
00185 Roma
See on map
75654 Paris
See on map
28006 Madrid
See on map
142 20 Praha 4
See on map
22419 Hamburg
See on map
The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.
Partners (10)
Partner organisations contribute to the implementation of the action, but do not sign the Grant Agreement.
8005 Zurich
See on map
Partner organisations contribute to the implementation of the action, but do not sign the Grant Agreement.
9052 Zwijnaarde
See on map
Partner organisations contribute to the implementation of the action, but do not sign the Grant Agreement.
See on map
Partner organisations contribute to the implementation of the action, but do not sign the Grant Agreement.
4051 Basel
See on map
Partner organisations contribute to the implementation of the action, but do not sign the Grant Agreement.
D02 Dublin
See on map
Partner organisations contribute to the implementation of the action, but do not sign the Grant Agreement.
6525 XZ Nijmegen
See on map
Partner organisations contribute to the implementation of the action, but do not sign the Grant Agreement.
81100 Caserta
See on map
Partner organisations contribute to the implementation of the action, but do not sign the Grant Agreement.
34090 Montpellier
See on map
Partner organisations contribute to the implementation of the action, but do not sign the Grant Agreement.
41013 Sevilla
See on map
Partner organisations contribute to the implementation of the action, but do not sign the Grant Agreement.
116 36 Praha 1
See on map