Patient-Derived Xenograft Models in Leukemia Research: Advancing Preclinical Studies

Leukemia research is limited by disease complexity and challenges in therapy development. Patient-derived xenograft (PDX) models mitigate these issues by preserving crucial patient-specific features. We expanded primary leukemia cells in NSG or MISTRG mice, alongside 12 established cell lines, and performed RNA sequencing (RNAseq) on patient samples, PDX models, and cell lines. A paired patient/PDX analysis revealed preserved gene expression fidelity in PDXs, while cell lines mirrored only certain patient transcriptomic signatures. The 55 generated PDX models reflected the main pediatric AML subtypes, including acute megakaryoblastic leukemia, KMT2A-rearrangement, myeloid leukemia of Down syndrome, t(8;21), inv(16), NUP98-rearrangement, and RUNX1 mutations. These findings confirm that PDX models closely recapitulate patient biology, enabling translational studies and bridging the gap between in vitro experimentation and clinical testing.

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Artikel online veröffentlicht:
09. Mai 2025