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Functional Genomics of the Lysosome

Objective

For a long time the lysosome has been viewed as a “static” organelle that performs “routine” work for the cell, mostly pertaining to degradation and recycling of cellular waste. My group has challenged this view and used a systems biology approach to discover that the lysosome is subject to a global transcriptional regulation, is able to adapt to environmental clues, and acts as a signalling hub to regulate cell homeostasis. Furthermore, an emerging role of the lysosome has been identified in many types of diseases, including the common neurodegenerative disorders Parkinson's and Alzheimer’s. These findings have opened entirely new fields of investigation on lysosomal biology, suggesting that there is a lot to be learned on the role of the lysosome in health and disease. The goal of LYSOSOMICS is to use “omics” approaches to study lysosomal function and its regulation in normal and pathological conditions. In this “organellar systems biology project” we plan to perform several types of genetic perturbations in three widely used cell lines and study their effects on lysosomal function using a set of newly developed cellular phenotypic assays. Moreover, we plan to identify lysosomal protein-protein interactions using a novel High Content FRET-based approach. Finally, we will use the CRISPR-Cas9 technology to generate a collection of cellular models for all lysosomal storage diseases, a group of severe inherited diseases often associated with early onset neurodegeneration. State-of-the-art computational approaches will be used to predict gene function and identify disease mechanisms potentially exploitable for therapeutic purposes. The physiological relevance of newly identified pathways will be validated by in vivo studies performed on selected genes by using medaka and mice as model systems. This study will allow us to gain a comprehensive understanding of lysosomal function and dysfunction and to use this knowledge to develop new therapeutic strategies.

Fields of science (EuroSciVoc)

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Programme(s)

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Topic(s)

Calls for proposals are divided into topics. A topic defines a specific subject or area for which applicants can submit proposals. The description of a topic comprises its specific scope and the expected impact of the funded project.

Funding Scheme

Funding scheme (or “Type of Action”) inside a programme with common features. It specifies: the scope of what is funded; the reimbursement rate; specific evaluation criteria to qualify for funding; and the use of simplified forms of costs like lump sums.

ERC-ADG - Advanced Grant

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Call for proposal

Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.

(opens in new window) ERC-2015-AdG

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Host institution

FONDAZIONE TELETHON ETS
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.

€ 2 362 562,50
Address
VIA VARESE 16/B
00185 ROMA
Italy

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Region
Centro (IT) Lazio Roma
Activity type
Research Organisations
Links
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.

€ 2 362 562,50

Beneficiaries (1)

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