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ZIDOMS SIGNED

Using Zebrafish as a novel tool to Improve the Diagnosis and Outcome of Marfan Syndrome

Total Cost €

EC-Contrib. €

Partnership

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ZIDOMS project word cloud

Explore the words cloud of the ZIDOMS project. It provides you a very rough idea of what is the project "ZIDOMS" about.

precision discovery clinical leads therapies personalized cure positive clinic mutations complications treatment rare model of classify prof evaluation mfs variants drug supportive expertise synergy researcher efficient screening symptoms nevertheless treatments zebrafish dr personal sips detection libraries fbn1 marfan cardiovascular syndrome fibrillin look identification animal proof lifelong deleterious linked classification unmet gene predictive classifications syndromic aid strategy disease disorder biological combination quick significance causal recent backer prevent relies physiology validate patients morbidity de medicine optimal models advantage supervisor patient diagnosis mortality mutation historical proposing genetic exists definitive spectrum unequivocally technological unknown tool potentially

Project "ZIDOMS" data sheet

The following table provides information about the project.

Coordinator	UNIVERSITEIT GENT Organization address address: SINT PIETERSNIEUWSTRAAT 25 city: GENT postcode: 9000 website: http://www.ugent.be contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.
Coordinator Country	Belgium [BE]
Total cost	172˙800 €
EC max contribution	172˙800 € (100%)
Programme	1. H2020-EU.1.3.2. (Nurturing excellence by means of cross-border and cross-sector mobility)
Code Call	H2020-MSCA-IF-2017
Funding Scheme	MSCA-IF-EF-RI
Starting year	2018
Duration (year-month-day)	from 2018-09-01 to 2020-08-31

Partnership

Take a look of project's partnership.

#	participants	country	role	EC contrib. [€]
1	UNIVERSITEIT GENT UNIVERSITEIT GENT Organization address address: SINT PIETERSNIEUWSTRAAT 25 city: GENT postcode: 9000 website: http://www.ugent.be contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	BE (GENT)	coordinator	172˙800.00

Map

Project objective

Recent technological advances have brought the implementation of personal and precision medicine in the clinic within reach. In this project, we are proposing to use zebrafish as a novel, efficient tool to improve the clinical diagnosis and treatment of Marfan Syndrome (MFS), a rare genetic disorder with a wide spectrum of clinical features. Since there is no definitive cure for MFS, patients need lifelong supportive treatment to prevent morbidity and mortality related to cardiovascular complications. Together with the detection of a number of syndromic symptoms, positive diagnosis of MFS relies on the identification of a deleterious mutation in the fibrillin-1 (FBN1) gene. Nevertheless, genetic testing often leads to the discovery of FBN1 variants which are not unequivocally linked to disease development. Current practice relies on the evaluation of historical and predictive parameters to classify variants of unknown significance as potentially causal mutations, but no biological model exists to efficiently validate the genetic classifications. This project aims to make optimal use of the combination of the know-how of the researcher, Dr. Sips, in the field of cardiovascular physiology and animal models of disease, with the clinical expertise of the supervisor, Prof. De Backer, in the diagnosis and treatment of MFS patients. The goal is to develop a zebrafish-based model that will allow for the quick classification of unknown variants in the FBN1 gene to aid in the diagnosis of MFS. In addition, the model will allow an efficient screening of drug libraries to look for potential new therapies, which might make it possible to find patient-specific treatments. Taken together, this project aims to take advantage of the synergy between the researcher and the supervisor in order to develop a novel strategy for a personalized approach to medicine. MFS is studied as a proof-of-concept clinical challenge with unmet needs in diagnosis and treatment.

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The information about "ZIDOMS" are provided by the European Opendata Portal: CORDIS opendata.