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Reprogramming cell identity to develop new therapies against Inherited Metabolic Disorders of the liver

Obiettivo

Liver diseases are becoming one of the most common causes of mortality in developing countries, and orthotopic liver transplantation is the only available treatment. However, this procedure carries implies indefinite immunosupression treatment associated with heavy side effects and eventual rejection. Furthermore, an increasing number of patients die while on the liver transplant waiting list due to the shortage of donor livers. Hepatocyte transplantation recently became an alternative to transplantation for the treatment of life-threatening Ineherithed Metabolic Diseases (IMDs) of the liver. However, this approach is also hampered by the lack of donors and by the difficulty in expending hepatocytes in vitro. Therefore, developing alternative source of hepatocytes represents a major challenge for the regenerative medicine field. Pluripotent stem cells generated from reprogrammed somatic cells (human Induced Pluripotent Stem Cells or hIPSCs) represent an advantageous solution since they can proliferate indefinitely in vitro while maintaining their capacity to differentiate into a broad number of cell types including hepatocytes. In addition, hIPSCs could enable the production of patient specific cells fully immuno-compatible with the original donor thereby avoiding the need for immune suppressive treatment. Here, we propose to systematically address the limitations preventing the use of hIPSCs for cells based therapy of IMDs. We will first develop novel methods to generate “better” hIPSCs fully compatible with clinical applications and to differentiate them into adult hepatocyes. In parallel, we will establish a novel approach for editing the mammalian genome and to correct the genetic defects associated with IMDs. Finally, we will validate the safety and the functionality of hIPSCs derived hepatocytes in vivo. Overall this comprehensive study will aim to provide the first proof of principle that hIPSCs could be useful in novel therapies against IMDs.

Invito a presentare proposte

ERC-2011-StG_20101109
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Meccanismo di finanziamento

ERC-SG - ERC Starting Grant

Istituzione ospitante

THE CHANCELLOR MASTERS AND SCHOLARS OF THE UNIVERSITY OF CAMBRIDGE
Contributo UE
€ 1 500 000,00
Indirizzo
TRINITY LANE THE OLD SCHOOLS
CB2 1TN Cambridge
Regno Unito

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Regione
East of England East Anglia Cambridgeshire CC
Tipo di attività
Higher or Secondary Education Establishments
Contatto amministrativo
Renata Schaeffer (Ms.)
Ricercatore principale
Ludovic Patrick Vallier (Dr.)
Collegamenti
Costo totale
Nessun dato

Beneficiari (1)