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Patient-derived xenograft models in cancer therapy: technologies and applications

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SPRINGERNATURE
DOI: 10.1038/s41392-023-01419-2

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Patient-derived xenograft (PDX) models, which involve implanting tumor tissues from patients into mice, have proven to be superior in replicating the characteristics of cancer. They retain the genomic features of patients and have enabled a comprehensive understanding of the molecular landscape of PDX models. PDX models are invaluable in cancer treatment studies, such as preclinical trials, drug validation, patient screening, and exploring drug resistance mechanisms.
Patient-derived xenograft (PDX) models, in which tumor tissues from patients are implanted into immunocompromised or humanized mice, have shown superiority in recapitulating the characteristics of cancer, such as the spatial structure of cancer and the intratumor heterogeneity of cancer. Moreover, PDX models retain the genomic features of patients across different stages, subtypes, and diversified treatment backgrounds. Optimized PDX engraftment procedures and modern technologies such as multi-omics and deep learning have enabled a more comprehensive depiction of the PDX molecular landscape and boosted the utilization of PDX models. These irreplaceable advantages make PDX models an ideal choice in cancer treatment studies, such as preclinical trials of novel drugs, validating novel drug combinations, screening drug-sensitive patients, and exploring drug resistance mechanisms. In this review, we gave an overview of the history of PDX models and the process of PDX model establishment. Subsequently, the review presents the strengths and weaknesses of PDX models and highlights the integration of novel technologies in PDX model research. Finally, we delineated the broad application of PDX models in chemotherapy, targeted therapy, immunotherapy, and other novel therapies.

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