ERYTHROTHERAPY

DEFINING NEW DRUGS AND DRUG TARGETS FOR TREATMENT OF ANEMIA

 Coordinatore LUNDS UNIVERSITET 

 Organization address address: Paradisgatan 5c
city: LUND
postcode: 22100

contact info
Titolo: Ms.
Nome: Stina
Cognome: Ahlenius
Email: send email
Telefono: +46 46 173465
Fax: +46 46 130064

 Nazionalità Coordinatore Sweden [SE]
 Totale costo 100˙000 €
 EC contributo 100˙000 €
 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-2011-CIG
 Funding Scheme MC-CIG
 Anno di inizio 2012
 Periodo (anno-mese-giorno) 2012-04-01   -   2016-03-31

 Partecipanti

# participant  country  role  EC contrib. [€] 
1    LUNDS UNIVERSITET

 Organization address address: Paradisgatan 5c
city: LUND
postcode: 22100

contact info
Titolo: Ms.
Nome: Stina
Cognome: Ahlenius
Email: send email
Telefono: +46 46 173465
Fax: +46 46 130064

SE (LUND) coordinator 100˙000.00

Mappa


 Word cloud

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

aim    rna    strategy    dba    epo    progenitor    treatment    cells    cell    drugs    patients    red    drug    interference    blood    rp    strategies    recombinant    anemia    mouse    genes    disease   

 Obiettivo del progetto (Objective)

'More than a billion people suffer from anemia. The only drug used to symptomatically increase red blood cell production in anemic patients is recombinant erythropoietin (Epo). While Epo injections effectively promote red blood cell production in patients suffering low endogenous Epo production, aplastic anemia and anemia associated with cancer, inflammation and infection do not respond well to recombinant Epo treatment.

Aim A: Since there is a great need for new drugs that can treat Epo-resistant anemias the first aim of ERYTHROTHERAPY is to identify novel drug target for production of Epo-responsive cells. Using next-generation mRNA sequencing we identified 250 candidate genes that have the potential to be targeted by drugs and are highly expressed in red blood cell progenitor cells. RNA interference based screening strategies will be used to systematically characterize which if these genes can be targeted to promote red blood cell progenitor proliferation. If successful this study will lead to development of a novel class of erythropoiesis stimulating agents able to manage conditions that today lack pharmacological treatment.

Aim B: The second goal of this project is to identify mechanism-based treatment strategies for Diamond-Blackfan anemia (DBA; OMIM #105650). All known DBA disease genes encode for ribosomal protein (RP) genes. Despite the recent advances in DBA genetics the pathophysiology remains elusive, which until now has prevented development of disease-specific therapies. In order to develop effective and specific DBA drugs the ERYTHROTHRAPY proposal uses an animal model for DBA to identify the molecular mechanisms linking RP-deficiency to anemia and bone marrow failure. Since limitations of previous mouse models for DBA make them unsuitable for this research strategy we generated a novel RP-deficient DBA mouse by taking advantage of Doxycycline-regulatable RNA interference. The drug-inducible strategy allows reversible and dose-dependent downregula'

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