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The role of protein degradation in FBXO11-related intellectual disability

Project description

Protein homeostasis during neurodevelopment

Neurodevelopmental disorders (NDDs) affect brain development and function and are characterised by genetic and phenotypic heterogeneity. Recent evidence underscores a role for aberrant protein homeostasis in NDDs. The EU-funded Degradation_ID project aims to provide insight into components of the protein homeostasis machinery. To achieve this, researchers will focus on FBXO11, which is known for its modification capacity of proteins destined for degradation. Mutations in FBXO11 have been linked to NDDs so further insight into the mechanism of FBXO11 function in neurodevelopment is necessary. Drosophila melanogaster will be used to model FBXO11 deficiency and screen various proteasome activators as a strategy for overcoming protein degradation impairment.

Objective

Neurodevelopmental disorders (NDDs) are genetically extremely heterogeneous with mutations in more than 1000 genes being causative. NDD-related genes and encoded proteins are involved in a wide range of various biological processes. Recently, several genes involved in protein degradation have been implicated in NDDs. This suggests that defects in protein homeostasis may be a recurring theme in neurodevelopment and cognition. We have recently identified de novo mutations in the nuclear E3-ubiquitin ligase complex component FBXO11 in 20 patients with a variable neurodevelopmental disorder. Loss-of function or haploinsufficiency of FBXO11 is the most likely mechanism for FBXO11-related NDDs. The pathomechanism of how FBXO11deficiency may lead to (neuro)-developmental defects has yet to be established. The objective of this project is to understand the role FBXO11 defects may play in the development of NDDs. In aim 1 I will focus on deciphering the molecular function of FBXO11 using two complementary approaches: identifying FBXO11 substrates using affinity-purification mass-spectrometry approaches and identifying pathways affected by FBXO11 dosage alterations using transcriptomic approaches. The identification of FBXO11 substrates and downstream pathways will lead to a better understanding of its role in neurodevelopment and in NDDs. In aim 2 I will characterize the role of Fbxo11 in neurodevelopment by modeling Fbxo11 deficiency in Drosophila melanogaster and investigate the potential to manipulate phenotypes by supplementation of fly food with proteasome activators. Collectively, this project will furthermore open a window to better understand the role alterations of protein degradation may play for NDDs in general.

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MSCA-IF-EF-RI - RI – Reintegration panel

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Call for proposal

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(opens in new window) H2020-MSCA-IF-2018

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Coordinator

UNIVERSITAET BERN
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.

€ 43 701,60
Address
HOCHSCHULSTRASSE 6
3012 BERN
Switzerland

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Region
Schweiz/Suisse/Svizzera Espace Mittelland Bern / Berne
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.

€ 43 701,68

Participants (1)

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