CRISPR/Cas9 in vivo screens in patients’ leukemias growing in mice

Functional genomic screens were mostly performed in cell lines and mouse models so far. To approximate the clinical situation, we established customized CRISPR/Cas9 screens in patient-derived xenograft (PDX) leukemia models in vivo. Ninety-two surface molecules were tested; sgRNAs against CXCR4 and ITGB1 dropped out and single knockouts validated their essential function. Our method allows determining patient-specific tumor dependencies in vivo which might facilitate personalized medicine of cancer in the future. Whole-Transcriptome analysis of 36 pediatric and adult patient-derived xenograft (PDX) acute lymphoblastic leukemia (ALL) samples. Samples were taken in the majority in the relapsed/refractory setting. *** Submitter states that German law does not allow raw data to be submitted for public access. The raw data of this study is available, but German law restrictions apply to the availability of these data, which were used for the current study, and so are not publicly available. Data are however available from the authors upon reasonable request and with permission of the local ethics committee.