Precision medicine is the new paradigm of modern cancer treatment and gives hope to an increasing number of cancer patients. Unfortunately, childhood cancers seem to lag behind this development mainly because they are rare and therefore difficult to study and they have little commercial interest. While rhabdomyosarcoma (RMS) has a high probability for positive treatment outcome, many patients with metastatic disease succumb to their disease and patients with relapse usually have a dismal prognosis. Furthermore, the high positive treatment rates in childhood cancer may be misleading as many pediatric cancer patients suffer from severe side effects later in life. It is therefore clear that new treatment strategies are required to improve the outcome of pediatric cancer patients.
CRISPR based technologies have revolutionized medical research in the last years and have opened up for affordable genetic screens in mammalian cells to investigate functional networks in cancer. We will perform genetic screens in selected rhabdomyosarcoma cell lines of both alveolar and embryonal origin in order to identify relevant targets for precision medicine. A focused library towards drug targets, kinases and phosphatases will be applied and the functional readout of the screen will be cell viability in order to identify targets that can inhibit the growth of RMS cells.
Novel targets from CRISPR screens will be validated by immunohistochemistry on RMS patient material from the Norwegian Childhood Cancer Biobank. Drugs against selected promising candidates will be tested in panels of RMS cell lines. Thus, we will identify novel targets for precision therapy in RMS with the possibility for further clinical testing.
Tagged as: Life Sciences
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