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Advances in the Diagnosis and Gene Therapy of Congenital dyserythropoietic Anemia

Project description

Novel tools for the diagnosis and treatment of congenital anemias

Congenital dyserythropoietic anemias (CDA) are a group of hereditary hematological diseases (HHD) characterised by congenital anemia with ineffective erythropoiesis. The scope of the EU-funded ADGENTHE-CDA project is to develop tools for the diagnosis and treatment of CDA focussing on state-of-the-art technologies. Scientists will identify novel genes and establish a genetic panel for CDA diagnosis using the revolutionary next-generation sequencing (NGS) technology. This will improve the opportunity for prompt treatment sparing patients from unwanted side effects. Through cellular CDA models and gene therapy tools, the project will pave the way for novel and more effective therapies.

Field of science

  • /medical and health sciences/medical biotechnology/genetic engineering/gene therapy

Call for proposal

See other projects for this call

Funding Scheme

MSCA-IF-EF-SE - Society and Enterprise panel
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Verge De Guadalupe 18
08950 Esplugues De Llobregat
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
EU contribution
€ 172 932,48