TARGETINGGENETHERAPY

Towards Safe and Effective Hematopoietic Stem Cell Gene Therapy: Targeting Integration to Genomic Safe Harbors and Exploiting Endogenous microRNA to Regulate Transgene Expression

 Coordinatore UNIVERSITA VITA-SALUTE SAN RAFFAELE 

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 Nazionalità Coordinatore Italy [IT]
 Totale costo 2˙500˙000 €
 EC contributo 2˙500˙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-2009-AdG
 Funding Scheme ERC-AG
 Anno di inizio 2010
 Periodo (anno-mese-giorno) 2010-05-01   -   2016-01-31

 Partecipanti

# participant  country  role  EC contrib. [€] 
1    Ospedale San Raffaele

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

contact info
Titolo: Dr.
Nome: Maria Rosa
Cognome: Pedrazzi
Email: send email
Telefono: +39 02 2643 4845
Fax: +39 02 2643 4717

IT (Milano) beneficiary 1˙096˙197.87
2    FONDAZIONE CENTRO SAN RAFFAELE DEL MONTE TABOR

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

contact info
Titolo: Mr.
Nome: Mario
Cognome: Valsecchi
Email: send email
Telefono: +39 02 26432729
Fax: +39 02 26432752

IT (MILANO) beneficiary 493˙002.13
3    UNIVERSITA VITA-SALUTE SAN RAFFAELE

 Organization address address: Via Olgettina 58
city: MILANO
postcode: 20132

contact info
Titolo: Dr.
Nome: Maria Rosa
Cognome: Pedrazzi
Email: send email
Telefono: +39 02 26434845
Fax: +39 02 2643 4717

IT (MILANO) hostInstitution 910˙800.00
4    UNIVERSITA VITA-SALUTE SAN RAFFAELE

 Organization address address: Via Olgettina 58
city: MILANO
postcode: 20132

contact info
Titolo: Prof.
Nome: Luigi
Cognome: Naldini
Email: send email
Telefono: +39 02 2643 4681
Fax: +39 02 2643 4621

IT (MILANO) hostInstitution 910˙800.00

Mappa


 Word cloud

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

finger    transgene    cells    medicine    hematopoietic    disease    stem    zinc    gene    vectors    clinical    expression    therapy    strategies    transfer    regulating   

 Obiettivo del progetto (Objective)

'Hematopoietic stem cell gene therapy has a tremendous potential to treat human disease. Yet, in conjunction with the first successful results in the clinic, severe adverse events linked to the gene transfer protocol were reported. Recently, we provided proof-of-principle of two new powerful strategies to improve the efficacy and safety of gene transfer: 1) regulating transgene expression by exploiting cellular microRNAs; 2) targeting integration at predetermined sites of the genome by forcing homologous recombination with designer Zinc finger nucleases. Here we will investigate the microRNA network regulating hematopoiesis and exploit the new knowledge to develop vectors with stringently controlled expression throughout the hematopoietic lineages. We will develop Zinc finger nuclease-based vectors that insert the transgene with high efficiency and specificity either downstream to its own endogenous promoter or into a safe genomic harbor that allows for robust expression without interference on the neighboring genes. By combining these strategies we will provide radically improved gene transfer platforms. Furthermore, we will exploit these technologies for the generation and genetic correction of induced pluripotent stem cells, providing a potentially unlimited source of patient-derived vector free gene corrected multipotent stem cells for future applications of regenerative medicine. The new gene therapy strategies will be tested in pre-clinical models of leukodystrophies and immunodeficiencies, for which we have extensive experience, and should enter a clinical trial for at least one such disease by the end of the proposed funding period. If successfully validated, the new strategies may eventually broaden the scope of gene therapy in medicine.'

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