CN-I LIVER THERAPY

POTENTIAL OF INDUCED PLURIPOTENT STEM CELLS FOR THE TREATMENT OF CRIGLER-NAJJAR LIVER DISEASE: A PRECLINICAL SAFETY ASSESSMENT

 Coordinatore UNIVERSITE DE GENEVE 

 Organization address address: Rue du General Dufour 24
city: GENEVE
postcode: 1211

contact info
Titolo: Prof.
Nome: Barbara
Cognome: Wildhaber
Email: send email
Telefono: +41 22 382 46 62

 Nazionalità Coordinatore Switzerland [CH]
 Totale costo 199˙317 €
 EC contributo 199˙317 €
 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-2013-IEF
 Funding Scheme MC-IEF
 Anno di inizio 2014
 Periodo (anno-mese-giorno) 2014-11-01   -   2016-10-31

 Partecipanti

# participant  country  role  EC contrib. [€] 
1    UNIVERSITE DE GENEVE

 Organization address address: Rue du General Dufour 24
city: GENEVE
postcode: 1211

contact info
Titolo: Prof.
Nome: Barbara
Cognome: Wildhaber
Email: send email
Telefono: +41 22 382 46 62

CH (GENEVE) coordinator 199˙317.60

Mappa


 Word cloud

Esplora la "nuvola delle parole (Word Cloud) per avere un'idea di massima del progetto.

metabolic    hep    stem    combination    differentiation    differentiated    cell    disease    transplantation    promising    therapy    gene    patient    ips    liver    lt    cni    reprogramming    safety    cn    epigenetic    corrected    cells    amplification   

 Obiettivo del progetto (Objective)

'Currently, the only cure for many inherited metabolic liver diseases with severe extra-hepatic manifestations, such as Crigler-Najjar disease (CNI), is liver transplantation (LT). LT goes together with heavy surgery, life-long immunosuppression and important public health costs. Development of less invasive treatments will permit important benefits for the patients and economic savings. Combination of gene therapy and hepatocyte (HEP) transplantation is a promising alternative to LT, correcting the metabolic defect while keeping in place the normal native liver. Nevertheless, the major obstacle has been shown to be the insufficient amount of corrected HEP available for transplantation. Induced pluripotent stem cells (iPS) are endowed with intrinsic self-renewal ability and the potential to differentiate into any of the three germ layers allowing cell amplification before differentiation. As such, iPS are heralded as the most promising avenue for cell-based therapeutics. However, recent evidences indicate that the epigenetic features of iPS are heterogeneous and notably influenced by the tissue of origin of the reprogrammed cells.

Hypothesis: HEP reprogramming and differentiation induce profound epigenetic changes that could affect the safety of gene and stem cell therapy combination approach.

Objectives: 1) Reprogramming of patient-specific CNI-HEP into iPS. 2) Correction of CNI-iPS by ex vivo lentiviral transduction. 3) Large-scale amplification of the corrected CNI-iPS and differentiation to HEP-like cells. 4) Epigenetic analysis of patient CNI-HEP, CN-iPS and the differentiated-corrected cells. 5) Therapeutic assessment of corrected CN-HEP-like cells into the liver of the CNI animal model.

This project will allow a step forward to studying the safety, efficacy and clinical potential of differentiated cells derived from patient-specific iPS. Importantly, not only CNI disease but also many other inborn liver disorders are potentially treatable via this approach.'

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