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
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