IPSMILD

HUMAN INDUCED PLURIPOTENT STEM CELLS AS A MODEL TO STUDY METABOLIC INHERITED LIVER DISEASES

Coordinatore	INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM)    more  Organization address address: 101 Rue de Tolbiac city: PARIS postcode: 75654 contact info Titolo: Ms. Nome: Fatima Cognome: Baghdali Email: send email Telefono: +33 2 40 35 86 79 Fax: +33 2 40 47 77 01
Nazionalità Coordinatore	France [FR]
Totale costo	75˙000 €
EC contributo	75˙000 €
Programma	FP7-PEOPLE Specific programme "People" implementing the Seventh Framework Programme of the European Community for research, technological development and demonstration activities (2007 to 2013)
Code Call	FP7-PEOPLE-2010-RG
Funding Scheme	MC-IRG
Anno di inizio	2011
Periodo (anno-mese-giorno)	2011-12-01   -   2014-11-30

 Partecipanti

#	participant	country	role	EC contrib. [€]
1	INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM)  Organization address address: 101 Rue de Tolbiac city: PARIS postcode: 75654 contact info Titolo: Ms. Nome: Fatima Cognome: Baghdali Email: send email Telefono: +33 2 40 35 86 79 Fax: +33 2 40 47 77 01	FR (PARIS)	coordinator	75˙000.00

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 Obiettivo del progetto (Objective)

'Familial hypercholesterolemia type IIa (FH) is a life-threatening disease caused by mutations on the Low Density Lipoprotein Receptor (LDLR). While drug treatments reduce the level of circulating cholesterol in heterozygote patients (1:500) with variable efficiency, homozygote patients (1:106) can only be treated by bimonthly apheresis, an invasive procedure. Hepatocytes are the only cells capable to internalize and degrade cholesterol. We previously shown that we were able to generated iPS from normal human fibroblasts and differentiated them into hepatocyte-like cells. Our aim is to develop preclinical tools and models to study human monogenic liver metabolic diseases and evaluate the ex vivo gene/cell therapy approaches. Our study will be focused on patient specific model for FH. In addition to reproducing the pathology in vitro with hepatocyte-like cells-derived from heterozygote and homozygote FH patients, this model will allow us to study the impact of LDLR mutations on cholesterol homeostasis in a human context. At a more long-term, FH-derived hepatocytes should represent alternative tools for drug screening in an actual current context in which there are no cellular models displaying features of hepatocytes. They will enable testing the efficacy of new drugs on cholesterol pathway and their effects on the specific disease-related hepatocyte phenotype (cell differentiation and proliferation). Finally, this model will provide a tool to evaluate autologous gene/cell therapy with iPS-derived hepatocyte-like cells.'