Projektbeschreibung
Europäisches Schulungsprogramm für autosomal dominante Netzhauterkrankungen
In Europa sind etwa 350 000 Menschen von erblichen Netzhauterkrankungen betroffen, einer Gruppe von Krankheiten, die früher als unheilbar galten. Jüngere Fortschritte haben jedoch bahnbrechende neue Behandlungsmethoden hervorgebracht. Zu den wesentlichen Hindernissen gehören ungeklärte genetische Diagnosen, die Unkenntnis der Krankheitsmechanismen und die Entwicklung einer Gentherapie für autosomal dominante erbliche Netzhauserkrankungen (adIRD), die 25-40 % aller derartigen Fälle ausmachen. Im Rahmen des über die Marie-Skłodowska-Curie-Maßnahmen finanzierten Projekts ProgRet wird ein europäisches Schulungsnetz mit besonderem Schwerpunkt auf den Mechanismen, der Diagnose und der Therapie von erblichen Netzhauterkrankungen eingerichtet. Die Projektziele umfassen die Verwendung von Stammzell- und Tiermodellen zur Untersuchung der adIRD-Mechanismen, eine bessere Diagnostik durch ein Einzelmolekül-Multiomik-Framework und die Entwicklung innovativer Behandlungen auf der Grundlage von RNA-Therapie und CRISPR-Genomeditierung.
Ziel
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.
Wissenschaftliches Gebiet
- 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
Schlüsselbegriffe
Programm/Programme
- HORIZON.1.2 - Marie Skłodowska-Curie Actions (MSCA) Main Programme
Aufforderung zur Vorschlagseinreichung
Andere Projekte für diesen Aufruf anzeigenFinanzierungsplan
HORIZON-TMA-MSCA-DN - HORIZON TMA MSCA Doctoral NetworksKoordinator
9000 Gent
Belgien