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European Training Program to Understand, Diagnose and Treat Autosomal Dominant Retinal Diseases

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.

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Programme(s)

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Topic(s)

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Funding Scheme

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HORIZON-TMA-MSCA-DN - HORIZON TMA MSCA Doctoral Networks

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Call for proposal

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(opens in new window) HORIZON-MSCA-2022-DN-01

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Coordinator

UNIVERSITEIT GENT
Net EU contribution

Net EU financial contribution. The sum of money that the participant receives, deducted by the EU contribution to its linked third party. It considers the distribution of the EU financial contribution between direct beneficiaries of the project and other types of participants, like third-party participants.

€ 525 240,00
Address
SINT PIETERSNIEUWSTRAAT 25
9000 Gent
Belgium

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Region
Vlaams Gewest Prov. Oost-Vlaanderen Arr. Gent
Activity type
Higher or Secondary Education Establishments
Links
Total cost

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No data

Participants (7)

Partners (10)

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