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Exploring the role of TFEB in exosome biology and in novel approaches for treatment of lysosomal diseases

Objective

Exosomes are small vesicles released to the extracellular environment by almost every cell type, with important functions in mediating intercellular communication in several physiological processes, including organism development, immune responses, neuronal communication and tissue repair. In addition, these vesicles have attracted much interest from the biomedical research community for their potential as biomarkers for diseases, therapeutic agents and vehicles for drug delivery. Despite this, little is known about the mechanism and molecular players involved in exosome biogenesis and secretion. The aim of this application is to expand our current knowledge on exosome biology and their therapeutic potential. Preliminary results suggest that Transcription Factor EB (TFEB), a master regulator of lysosomal biogenesis, lysosomal exocytosis and autophagy induction, also controls the expression of various exosome-associated genes. By using mouse and human cells upregulated or knocked-down for TFEB expression, the applicant will establish a role for TFEB in exosome biogenesis, cargo selectivity and secretion. Furthermore, the applicant will determine the role of other TFEB-related cellular pathways, including starvation and Ca2+-related pathways, in regulating exosome formation. Finally, the applicant will explore the therapeutic potential of exosomes in cellular and mouse models of lysosomal storage disorders (LSDs). Collectively, this application combines both basic research and translational approaches to expand our current understanding of exosome biology and to provide proof of principle for novel therapeutic approaches of rare diseases.

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.

MSCA-IF-EF-ST - Standard EF

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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) H2020-MSCA-IF-2014

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Coordinator

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.

€ 180 277,20
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.

€ 180 277,20
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