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