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Identifying neuronal or muscle cell-specific suppressors that rescue motility defects in the ALS model of the nematode worm Caenorhabditis elegans

Project description

Identifying potential therapeutic targets in the ALS model of the Caenorhabditis elegans

Amyotrophic lateral sclerosis (ALS) is a fatal disease with progressive loss of neuronal and muscle function, for which there is currently no cure. The cytoplasmic accumulation of the TDP-43 protein in neurons is consistent among ALS patients, implying that it acts as a point of convergence in the pathway responsible for the disease's progression. The EU-funded RescueMoDe project aims to identify neuronal or muscle cell-specific suppressors of motor impairment using an ALS model of the nematode Caenorhabditis elegans. The transgenic C. elegans model overexpresses TDP-43 in the neurons, resulting in severe motility defects and enabling the analysis of the tissue-specific role of these suppressors. The study will provide a starting point for further functional ALS studies in mammalian cell culture and murine model systems, leading to an in-depth understanding of ALS progression.

Objective

Amyotrophic lateral sclerosis (ALS) is a fatal disease that progressively causes loss of neuronal and muscle function, for which there is no known cure. Although the genetic causes of ALS vary, the cytoplasmic accumulation of the TDP-43 protein in neurons is highly consistent among patients. Thus, TDP-43 is believed to be a point of convergence in the pathway responsible for ALS progression. However, while many genetic and cellular mechanisms have been linked to ALS, there is still a lack of understanding of the neuro-muscular interactions in ALS. In this project, we will identify neuronal or muscle cell-specific suppressors of motor impairment using an ALS model in the nematode worm Caenorhabditis elegans. In this model, transgenic C. elegans overexpress TDP-43 in the neurons, resulting in severe motility defects. We will use optogenetic tools to excite neurons and muscle cells separately in the C. elegans ALS model, contributing to our understanding of how TDP-43 accumulation affects tissue function. In addition, live in vivo microscopy of C. elegans will help us to elucidate the impact of TDP-43 on neuro-muscular interactions over time. Furthermore, novel automated tracking of the nematode worms enables high-throughput analysis of C. elegans mobility. Thus, we can efficiently analyse mobility when TDP-43 is overexpressed, and use this tracking for high-throughput screening of mutants that rescue the ALS phenotype. Once we have identified the mutants that Rescue Motility Defects (RescueMoDe), we will characterize their impact on neuronal and muscular function. Therefore, it will be possible to analyse the tissue-specific role of these candidates, and how they fit into the progression of TDP-43 toxicity in this system. Overall, we aim to further the understanding of ALS progression, which will allow a highly informed continuation of studies in mammalian cell culture or in murine model systems, which may lead to therapeutic research opportunities.

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Programme(s)

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Topic(s)

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Funding Scheme

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MSCA-IF - Marie Skłodowska-Curie Individual Fellowships (IF)

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Call for proposal

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(opens in new window) H2020-MSCA-IF-2019

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Coordinator

ACADEMISCH ZIEKENHUIS GRONINGEN
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.

€ 175 572,48
Address
HANZEPLEIN 1
9713 GZ Groningen
Netherlands

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Activity type
Higher or Secondary Education Establishments
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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.

€ 175 572,48
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