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Identifying Metabolic dysregulation to Guide Novel FOXP-ID/ASD Therapies

Project description

Exploring metabolic dysregulation in neurodevelopmental disorders

Neurodevelopmental disorders (NDDs) comprise a group of neurological and psychiatric conditions that disrupt normal brain development and function. Gene variants of FOXP1/2/4 transcription factors give rise to related NDDs variably affecting speech, cognition, and motor function. With the support of the Marie Skłodowska-Curie Actions programme, the IMAGiN-IT project will work under the hypothesis that metabolic dysregulation could critically contribute to the pathophysiology of FOXP-related NDDs. Using the highly efficient model organism Drosophila melanogaster, the research team will characterise clinically relevant behaviours and develop new strategies for image-based exploration of metabolic dysfunction in the brain. These insights will be used to develop intervention strategies that can ameliorate FOXP-associated phenotypes as a translational road towards treatment.

Objective

The mechanisms underlying monogenic intellectual disability (ID) and autism spectrum disorder (ASD), affecting >2% of the population, remain poorly understood. Moreover, most ID/ASD syndromes are still untreatable, the exception being some disorders of metabolic origin. Therefore, identifying unappreciated metabolic mechanisms underlying the pathophysiology of ID/ASD provides a promising avenue to treatment.

Several lines of evidence suggest that the transcription factors FOXP1/2/4 are ID/ASD genes with such an underappreciated role in brain (energy) metabolism: mitochondrial defects and altered metabolic rate in FOXP animal models, unpublished FoxP-/- brain transcriptome data of my host, and GWAS-based data indicating that FOXP1/2 genes are significantly associated with body mass index in the general population. Therefore, my main aim is to characterize ID/ASD phenotype-relevant metabolic dysregulation in FoxP mutants and ameliorate its effects. Powerful genetic tools in Drosophila allow me to probe specific molecular mechanisms with high spatial and temporal resolution and relate them to clinically relevant phenotypes, including locomotion behaviour habituation learning, and sleep. My objectives are to: 1) Identify phenotype-relevant metabolic target pathways of FoxP, 2) Characterize metabolic dysregulation in FoxP neurons with spatial resolution 3) Reverse behavioural FoxP phenotypes with metabolic or other innovative therapeutic interventions.

This interdisciplinary approach further develops the data analyses, molecular, and imaging skills that I acquired through my PhD training and allows me to merge it with unique disease-relevant data, clinical knowledge, and collaborating networks. This project has the potential to provide novel insights into the relation of metabolism, cognition, and behaviour, and revolutionize the treatment of FOXP1/2/4 disorders. These insights may also have implications for further molecularly and clinically related ID/ASD disorders.

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HORIZON-TMA-MSCA-PF-EF - HORIZON TMA MSCA Postdoctoral Fellowships - European Fellowships

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Call for proposal

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(opens in new window) HORIZON-MSCA-2023-PF-01

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Coordinator

STICHTING RADBOUD UNIVERSITAIR MEDISCH CENTRUM
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.

€ 203 464,32
Address
GEERT GROOTEPLEIN 10 ZUID
6525 GA NIJMEGEN
Netherlands

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Region
Oost-Nederland Gelderland Arnhem/Nijmegen
Activity type
Higher or Secondary Education Establishments
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Total cost

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