REGENERATIVETHERAPY

Cell and gene therapy approaches for inherited diseases with unsatisfying or no therapeutic option

Coordinatore	INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM)    more Spiacenti, non ci sono informazioni su questo coordinatore. Contattare Fabio per maggiori infomrazioni, grazie.
Nazionalità Coordinatore	France [FR]
Totale costo	2˙397˙215 €
EC contributo	2˙397˙215 €
Programma	FP7-IDEAS-ERC Specific programme: "Ideas" implementing the Seventh Framework Programme of the European Community for research, technological development and demonstration activities (2007 to 2013)
Code Call	ERC-2010-AdG_20100317
Funding Scheme	ERC-AG
Anno di inizio	2011
Periodo (anno-mese-giorno)	2011-10-01   -   2016-09-30

 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: Tiphaine Cognome: Guida Email: send email Telefono: 33140784913 Fax: 33140784998	FR (PARIS)	hostInstitution	2˙397˙215.00
2	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: Prof. Nome: Marina Cognome: Cavazzana-Calvo Email: send email Telefono: +33 1 44495068 Fax: +33 1 44492505	FR (PARIS)	hostInstitution	2˙397˙215.00

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 Obiettivo del progetto (Objective)

'We have developed the tools and experimental models and have provided the proof of principle that gene modified autologous haematopoietic stem cells can restore a complete immunological system in vivo in a particular form of SCID. We are pursuing these efforts applying this strategy to several other diseases : two lentivirus protocols for X-linked adrenoleukodystrophy, Thalassemia and sickle cell disease, are developed in our Clinical Investigation Centre. In 2010, we plan to initiate two other gene therapy trials for two primary immunodeficiencies (SCID-X1 and Wiskott-Aldrich syndrome). Our expertise in translational medicine has now been used to transfer into clinics a new gene therapy protocol for a devastating inherited skin disease : dystrophic epidermolysis bullosa. To complement these approaches, we optimize HLA-mismatched haematopoietic stem cell transplantation (HSCT). They are an important therapeutic option for children with primary immunodeficiencies but the delayed reconstitution of the T-cell compartment following T-cell depleted HSCT remains a major clinical concern thus drastically limiting its broader clinical application. In order to speed-up the immunological reconstitution in this setting, we try to pre-clinically develop a new approach able to generate a large quantity of T-cellular precursors from a fraction of the donor CD34 stem cells shortly cultured on Notch delta 4 ligand. This strategy could ideally provide the recipient with a pool of diverse T-cells within the first month after transplantation conferring T-cellular immunity before de novo thymopoiesis takes place. The results obtained with this procedure are so important that we think to extend its use to the transduction of HSC for the gene therapy trials.'