ArticleCancer discovery2021
A Humanized Animal Model Predicts Clonal Evolution and Therapeutic Vulnerabilities in Myeloproliferative Neoplasms.
Article in Cancer discovery, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 26 papers.
What it found
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Who cites it
26 citing papers in PubMed, 29 citations in OpenAlex.
- Menin-dependent megakaryocyte proliferation and fibrosis in myeloproliferative neoplasms.Cancer cell · 2026Article
- Establishing human immune system mice: application, opportunity, and challenges.Experimental & molecular medicine · 2026Review
- Engineering bone marrow in a dish-a bloody business: preclinical opportunities, translational use cases, and a call for consensus.Journal of thrombosis and haemostasis : JTH · 2026Review
- Advancing cancer research: Cutting-edge insights from colorectal cancer patient-derived xenograft mouse models.Genes & diseases · 2026Review
- Parallel Evolution of Leukemic Clones in Myeloproliferative Neoplasms.Research square · 2025Article
- Article
- Mesenchymal stromal cells fromHemaSphere · 2025Article
- Understanding Human Oncogene Function and Cooperativity in Myeloid Malignancy Using iPSCs.Experimental hematology · 2025Review
- RSK1 is an exploitable dependency in myeloproliferative neoplasms and secondary acute myeloid leukemia.Nature communications · 2025Article
- Application of omics in the diagnosis, prognosis, and treatment of acute myeloid leukemia.Biomarker research · 2024Review
- In vivo ablation of NF-κB cascade effectors alleviates disease burden in myeloproliferative neoplasms.Blood · 2024Article
- Establishment of a humanized mouse model using steady-state peripheral blood-derived hematopoietic stem and progenitor cells facilitates screening of cancer-targeted T-cell repertoires.Cancer innovation · 2024Article
- Generation of complex bone marrow organoids from human induced pluripotent stem cells.Nature methods · 2024Article
- Altered erythropoiesis via JAK2 and ASXL1 mutations in myeloproliferative neoplasms.Experimental hematology · 2024Article
- Engineering a humanized animal model of polycythemia vera with minimalHaematologica · 2024Article
- Patient-derived tumor models: a suitable tool for preclinical studies on esophageal cancer.Cancer gene therapy · 2023Review
- Progress in building clinically relevant patient-derived tumor xenograft models for cancer research.Animal models and experimental medicine · 2023Review
- Single-cell multi-omics identifies chronic inflammation as a driver of TP53-mutant leukemic evolution.Nature genetics · 2023Article
- Comprehensive profiling of clinical JAK inhibitors in myeloproliferative neoplasms.American journal of hematology · 2023Article
- Phenotypic characterization of disease-initiating stem cells in JAK2- or CALR-mutated myeloproliferative neoplasms.American journal of hematology · 2023Article
Corrections and comments
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Authors and funding
17 authors at 3 institutions in 1 country.
Funding
Abstract
Myeloproliferative neoplasms (MPN) are chronic blood diseases with significant morbidity and mortality. Although sequencing studies have elucidated the genetic mutations that drive these diseases, MPNs remain largely incurable with a significant proportion of patients progressing to rapidly fatal secondary acute myeloid leukemia (sAML). Therapeutic discovery has been hampered by the inability of genetically engineered mouse models to generate key human pathologies such as bone marrow fibrosis. To circumvent these limitations, here we present a humanized animal model of myelofibrosis (MF) patient-derived xenografts (PDX). These PDXs robustly engrafted patient cells that recapitulated the patient's genetic hierarchy and pathologies such as reticulin fibrosis and propagation of MPN-initiating stem cells. The model can select for engraftment of rare leukemic subclones to identify patients with MF at risk for sAML transformation and can be used as a platform for genetic target validation and therapeutic discovery. We present a novel but generalizable model to study human MPN biology. SIGNIFICANCE: Although the genetic events driving MPNs are well defined, therapeutic discovery has been hampered by the inability of murine models to replicate key patient pathologies. Here, we present a PDX system to model human myelofibrosis that reproduces human pathologies and is amenable to genetic and pharmacologic manipulation. This article is highlighted in the In This Issue feature, p. 2945.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.