Immune System
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Identification of novel immunotherapeutic targets in AML/MDS with spliceosome mutations using single cell long-read sequencing

Institution: Charité – Universitätsmedizin Berlin
Applicant: Dr. Livius Penter
Funding line:
First and Second Applications
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Acute myeloid leukemia (AML) is a difficult-to-treat blood malignancy that often relapses despite intensive therapy. Immunotherapies can contribute to preventing relapses. The key to developing novel immunotherapies is the identification of antigens uniquely presented by leukemia cells, enabling their specific elimination by immune cells (T cells). In AML with spliceosome mutations, altered mRNA processing (aberrant splicing) leads to the synthesis and presentation of abnormal antigens (neoepitopes). The scientists employ new sequencing tools such as long-read sequencing to elucidate aberrant splicing at the single-cell level, with the long-term goal of developing new T cell therapies.

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