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Targeting rogue DNA repair and genomic instability as novel toxicity mechanisms in C9orf72 ALS/FTD

Objective

Repeat expansions are a major cause of neurodegenerative diseases, but causal therapies are not available. A (G4C2)n repeat expansion in C9orf72 is the most common genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) in Europe. Despite extensive research, the mechanisms linking these expansions to selective neurodegeneration remain poorly understood. Our project, epiC9repair, explores the novel hypothesis that genomic instability at the C9orf72 locus, driven by aberrant DNA repair and repeat-induced chromatin changes, is a critical factor in ALS/FTD pathogenesis. This project builds on our recent observation that the C9orf72 repeat is also a hotspot of DNA synthesis in post-mitotic neurons, and on recent findings in Huntington’s disease that cell type-specific somatic expansion of the pathogenic (CAG)n repeat drives neurodegeneration by unknown mechanisms.
Using advanced CRISPR screens and innovative chromatin mapping techniques, we aim to uncover the DNA repair pathways that drive genomic instability in C9orf72 ALS/FTD and how these pathways contribute to neurodegeneration. In addition, we will investigate the role of epigenetic modifications in cell culture models and patient tissues, with a focus on identifying biomarkers that correlate with disease progression and regional neurodegeneration. These findings will inform studies in mouse models to test the therapeutic potential of targeting genomic instability. This multidisciplinary approach has the potential to transform our understanding of C9orf72-related diseases and paves the way for targeted therapeutic strategies that address the root causes of genomic instability in neurodegeneration.

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Keywords

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Programme(s)

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Topic(s)

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HORIZON-ERC - HORIZON ERC Grants

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Call for proposal

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(opens in new window) ERC-2024-ADG

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Host institution

DEUTSCHES ZENTRUM FUR NEURODEGENERATIVE ERKRANKUNGEN EV
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.

€ 2 498 905,00
Address
VENUSBERG-CAMPUS 1/99
53127 BONN
Germany

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Region
Nordrhein-Westfalen Köln Bonn, Kreisfreie Stadt
Activity type
Research Organisations
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Total cost

The total costs incurred by this organisation to participate in the project, including direct and indirect costs. This amount is a subset of the overall project budget.

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Beneficiaries (1)

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