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Ex-vivo screening platform to identify tumor-specific therapies

ABSTRACT: Ex-vivo screening platform to identify tumor-specific therapies

PROVIDER: PRJNA417676 | ENA |

REPOSITORIES: ENA

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Ex-vivo screening platform to identify tumor-specific therapies

Project description:We report the generation of a preclinical IBC patient-derived xenograft (PDX)-derived ex vivo tumor tissue model and show that it closely replicates the tissue architecture of the original PDX tumor harvested from mice and show that its genetic signature highly correlates with that of the original tumor. We used microarrays to evaluate the robustness and reproducibility of the method used to generate the ex vivo tumor tissue model and confirm its ability to recapitulate the essential features of the original tumor.

2018-04-07 | GSE106685 | GEO

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Ex Vivo Lung Perfusion as a Human Platform for Preclinical Small Molecule Testing

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An ex vivo physiologic and hyperplastic vessel culture model to study intra-arterial stent therapies

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A cyanobacterial screening platform for mutant variants of a Form II Rubisco

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Development of a split-toxin CRISPR screening platform to systematically identify upstream regulators of human myoblast fusion

Project description:Muscle defects are a common feature in human developmental disorders and often lead to severe functional impairment. These defects arise from intricate tissue crosstalk and rare genetic mutations, underscoring the need to systematically identify cell-autonomous mechanisms regulating human myogenesis. Despite the clinical significance, our understanding of human development remains limited, due in part to the absence of a scalable genetic approach to study this process. Here, we introduce a rationally designed high-throughput CRISPR screening platform that integrates human myoblast models, muscle-specific CRISPR knockout libraries, and a split-toxin strategy that acts as a functional readout for myoblast fusion—a fundamental step of human myogenesis. This screening strategy enables selection of CRISPR-induced fusion-defective myocytes in a quantifiable manner. Leveraging this platform, our initial genetic screen uncovered a large group of new hits essential for human myoblast fusion. The majority of these hits converge into 23 protein complexes, most of which have not previously been functionally linked to myogenesis in any species. Notably, mutations in 41 of our fusion screen hits cause human diseases presenting abnormal skeletal muscle morphology. Applying a new single-cell CRISPR & RNA-seq approach, we show that majority of these hits control human myoblast fusion as well as influence early-stage myogenic differentiation. Together, this work presents a new systematic approach to study human myogenesis and uncovers promising candidates governing human muscle differentiation and fusion. A broader application of this split-toxin based CRISPR screening platform would accelerate the study of cell-autonomous mechanisms of human muscle development and diseases at scale.

2025-10-24 | GSE293514 | GEO

Human Ex Vivo Lung Perfusion: A Model System to study Lung diseases and direct targeted therapies

Project description:Human Ex Vivo Lung Perfusion: A Model System to study Lung diseases and direct targeted therapies

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Proteogenomic subtyping of acute myeloid leukemia and ex-vivo drug response profiling for precision therapies.

Project description:We have performed a comprehensive proteomic analysis of clinical patient samples of acute myeloid leukemia (AML) alongside genome-, transcriptome- and ex-vivo drug response profiling.

2026-03-23 | PXD061758 | Pride

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