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Targeting the transcriptional landscape in infant AML

Periodic Reporting for period 3 - iAML-lncTARGET (Targeting the transcriptional landscape in infant AML)

Reporting period: 2019-07-01 to 2020-12-31

Infants with blood cancer particularly suffer from the side effects of the necessary intensive chemotherapy and have a poor prognosis. This highlights the need for new and innovative treatment approaches. The iAML-lncTARGET research team and others have recently revealed the importance of areas in the genome for the development of blood cancer that were previously considered as “junk”. These areas are extremely large and produce a variety of long and short non-coding RNAs. The production of these RNAs is extremely cell type-specific. Therefore those RNAs can serve as unique therapeutic targets. Still, the identification of relevant and novel targetable RNAs is technically extremely difficult. We will investigate those areas and evaluate therapeutic options to overcome current obstacles in the treatment of infants with blood cancer.
In our work, we highlighted the importance of small regulators – so called micro RNAs – for the diagnosis and progression of cancer. We could show that these small regulators could be used to predict the patient outcome and that manipulating those small regulators eradicated leukemia in relevant disease models. Further, we used this knowledge on micro RNAs on long RNAs to design and test novel therapeutic options in infants with high-risk leukemia. We identified several long RNAs that are involved in the pathogenesis of leukemia and can serve as therapeutic targets or biomarkers for leukemia. We think that our work paves the way for a better understanding and treatment of the fatal disease in infants.
The involvement of several of the identified non-coding RNAs in the pathogenesis and progression in blood cancer has not been described before. Hence, testing their future therapeutic potential and specificity goes beyond the start of the art and is an ambitious aim until the end of the project.
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