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Bioengineering of Humanized Bone Marrow Microenvironments in Mouse and Their Visualization by Live Imaging
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Understanding Normal and Malignant Human Hematopoiesis Using Next-Generation Humanized Mice.

Yoriko Saito1, Leonard D Shultz2, Fumihiko Ishikawa1

  • 1RIKEN Center for Integrative Medical Sciences, Yokohama City, Kanagawa, 230-0045, Japan.

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|July 8, 2020
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Summary

Humanized mice, which engraft human immune cells, offer advanced in vivo preclinical models for studying human diseases. These models aid in drug development and enable functional precision medicine through patient-derived xenografts and single-cell genomics.

Keywords:
human hematopoiesishuman immunityhumanized mouseleukemiaxenograft

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Area of Science:

  • Translational Medicine
  • Immunology
  • Preclinical Research

Background:

  • Rodent models are vital for understanding human physiology and disease.
  • Accelerated drug development necessitates improved in vivo preclinical models.
  • Humanized mice bridge the gap between traditional animal models and clinical applications.

Purpose of the Study:

  • To highlight the utility of humanized mice as advanced preclinical models.
  • To demonstrate their role in functional characterization of human cells in vivo.
  • To showcase their application in functional precision medicine.

Main Methods:

  • Development of immunodeficient mouse strains for high-level engraftment of human immune/hematopoietic cells.
  • Utilizing humanized mice as patient-derived xenograft (PDX) models.
  • Integration of single-cell genomics with humanized mouse models.

Main Results:

  • Humanized mice enable in vivo functional characterization of human immune/hematopoietic cells.
  • Patient-derived xenograft (PDX) models in humanized mice correlate mechanisms with in vivo behavior.
  • These models facilitate the revelation of pathogenic mechanisms.

Conclusions:

  • Humanized mice are crucial tools for advancing preclinical research and drug development.
  • They enable functional precision medicine, including risk stratification and personalized therapeutics.
  • Combining humanized mice with single-cell genomics offers powerful insights into human disease.