STEM CELLS FOR DMD

Novel strategies for the cell therapy of muscular dystrophies

 Coordinatore THE UNIVERSITY OF MANCHESTER 

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 Nazionalità Coordinatore United Kingdom [UK]
 Totale costo 1˙900˙000 €
 EC contributo 1˙900˙000 €
 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-2008-AdG
 Funding Scheme ERC-AG
 Anno di inizio 2009
 Periodo (anno-mese-giorno) 2009-03-01   -   2014-02-28

 Partecipanti

# participant  country  role  EC contrib. [€] 
1    FONDAZIONE CENTRO SAN RAFFAELE DEL MONTE TABOR

 Organization address address: Via Olgettina 60
city: MILANO
postcode: 20132

contact info
Titolo: Dr.
Nome: Massimiliano
Cognome: Meoni
Email: send email
Telefono: +39 02 26435480
Fax: +39 02 26434717

IT (MILANO) beneficiary 982˙240.18
2    UNIVERSITY COLLEGE LONDON

 Organization address address: GOWER STREET
city: LONDON
postcode: WC1E 6BT

contact info
Titolo: Ms.
Nome: Greta
Cognome: Borg-Carbott
Email: send email
Telefono: +44 2031083033
Fax: +44 20 78132849

UK (LONDON) beneficiary 591˙577.81
3    THE UNIVERSITY OF MANCHESTER

 Organization address address: OXFORD ROAD
city: MANCHESTER
postcode: M13 9PL

contact info
Titolo: Ms.
Nome: Claire
Cognome: Faichnie
Email: send email
Telefono: +44 161 275 1413

UK (MANCHESTER) hostInstitution 326˙182.01
4    THE UNIVERSITY OF MANCHESTER

 Organization address address: OXFORD ROAD
city: MANCHESTER
postcode: M13 9PL

contact info
Titolo: Prof.
Nome: Giulio
Cognome: Cossu
Email: send email
Telefono: 447446000000
Fax: 441613000000

UK (MANCHESTER) hostInstitution 326˙182.01

Mappa


 Word cloud

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

dogs    dmd    monitored    transduced    chromosome    reversibly    floxed    human    transfer    tert    clinical    dystrophin    cells    selected    transplanted    expressing    strategy    functional    artificial    bmi    mesoangioblasts    dystrophic    immune    clones    expression   

 Obiettivo del progetto (Objective)

This project aims to develop an effective cell therapy for Duchenne Muscular Dystrophy (DMD). To reach this goal we plan to transfer a human artificial chromosome containing the whole dystrophin locus into dystrophic mesoangioblasts. As the transfer is inefficient and requires selection, it is impossible to use primary cells that would undergo senescence during selection. Therefore we need to reversibly immortalize DMD patient mesoangioblasts by the combined use of lentiviral vectors expressing either floxed human telomerase (h-Tert) or floxed Bmi1 (and also the Herpes Simplex Thymidine Kinase, HSTK). Immortal clones, selected for proper expression of h-Tert and Bmi1 and maintenance of a mesoangioblast phenotype, will be transfected with a human artificial chromosome expressing human dystrophin. Expressing clones will be selected and characterized. Before transplantation, selected clones will be infected with an adenoviral vector expressing the Cre recombinase and then treated with Gancyclovir. Transduced and selected cells will be transplanted into mdx/SCID (dystrophic and immune deficient) mice and the extent of dystrophin reconstitution and consequent functional benefit will be evaluated. In addition, the possible immune reaction to the transgenes or novel antigens generated by the experimental strategy will be monitored by an in vitro assay utilizing donor s dendritic cells and T lymphocytes. Finally this strategy will be repeated in Golden Retriever dystrophic dogs, the closest animal model to DMD. Dog mesoangioblasts will be reversibly immortalized, transduced with the dystrophin HAC and, after viral excision and GC selection, transplanted in the same dogs from which they had been directly isolated. Dystrophin expression, functional amelioration and possible toxic and immune reactions will be monitored. This project will therefore cover all pre-clinical experimentation and would set the stage for immediate clinical translation.

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