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Structural and cellular investigation of the regulation of ATP8B1/CDC50A, a human flippase important for the hepatic function

Project description

The role of phospholipid transfer in cholestasis in children

Inherited intrahepatic cholestasis is a rare condition that affects children and is characterised by recurrent jaundice and failure to grow. Recently, mutations of the human lipid flippase ATP8B1 have emerged as a potential disease cause. The EU-funded LivFlip project will study the role of the ATP8B1 protein known to mediate the transfer of phospholipids to the cytoplasmic part of the membrane and maintain membrane asymmetry in eukaryotic cells. Insight into the biochemical basis of lipid selectivity in lipid flippases will help understand the functional and structural consequences of ATP8B1 mutations and their role in intrahepatic cholestasis.

Objective

Lipid flippases mediate transport of phospholipids from the exoplasmic leaflet to the cytosolic one, and their role is to establish and maintain phospholipid asymmetry in eukaryotic membranes. This asymmetry is tightly regulated and it is implicated in numerous cellular processes such as membrane trafficking, signaling, blood coagulation, apoptosis, cytokinesis, or cell fusion. The human genome encodes 14 different P4-ATPases, a subset of them being linked to pathologies such as mental retardation, Alzheimer’s disease, diabetes, cancer or liver disorders. As concerns the latter disease, it has been demonstrated that mutations of the human lipid flippase ATP8B1, are responsible for inherited intrahepatic cholestasis. The intrahepatic cholestasis affects mostly children and is characterized by pruritus, recurrent jaundice, growth failure and an evolutive liver degradation which requires transplantation before adulthood in most severe cases. Up to now, little is known about the function of ATP8B1 at the canalicular membrane and this is mainly due to the lack of consensus regarding its transport substrate and how its activity is regulated. Preliminary results indicate that ATP8B1 has a broader transport substrate specificity than previously reported in the literature and therefore its study will widen our understanding of the biochemical basis of lipid selectivity in lipid flippases. In addition, in the proposed project I aim at understanding how a disease related lipid flippase, ATP8B1, is regulated in vitro and in vivo and at understanding functional and structural consequences of identified mutations of ATP8B1 linked to the intrahepatic cholestasis.

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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-2020

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Coordinator

AARHUS UNIVERSITET
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.

€ 219 312,00
Address
NORDRE RINGGADE 1
8000 Aarhus C
Denmark

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Region
Danmark Midtjylland Østjylland
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.

€ 219 312,00
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