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The IDEAL project aims to test a specific inhibitor of DUX4-IGH, a genetic factor involved in B-cell acute lymphoblastic leukaemia, in pre-clinical models. The research seeks to demonstrate the inhibitor's effectiveness in blocking cancer cell transformation and improving treatment outcomes for affected children.
The second most important cause of death and morbidity in Europe is cancer and B-cell acute lymphoblastic leukaemia (B-ALL) is the most common paediatric cancer and cause of cancer-related death before 20 years.
In up to 7% of cases, the disease is caused by rearrangements of the genetic regulator DUX4 to the IGH locus, giving rise to the DUX4-IGH fusion.
While ectopic expression of DUX4 induces cell death, DUX4-IGH drives transformation.
Even though the two proteins share the same DNA binding d…
OSPEDALE SAN RAFFAELE SRL
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