Abstract

Mice xenotransplanted with human cells and/or expressing human gene products (also known as “humanized mice”) recapitulate the human evolutionary specialization and diversity of genotypic and phenotypic traits. These models can provide a relevant in vivo context for understanding of human‐specific physiology and pathologies. Humanized mice have advanced toward mainstream preclinical models and are now at the forefront of biomedical research. Here, we considered innovations and challenges regarding the reconstitution of human immunity and human tissues, modeling of human infections and cancer, and the use of humanized mice for testing drugs or regenerative therapy products. As the number of publications exploring different facets of humanized mouse models has steadily increased in past years, it is becoming evident that standardized reporting is needed in the field. Therefore, an international community‐driven resource called “Minimal Information for Standardization of Humanized Mice” (MISHUM) has been created for the purpose of enhancing rigor and reproducibility of studies in the field. Within MISHUM, we propose comprehensive guidelines for reporting critical information generated using humanized mice.

Highlights

  • Mice xenotransplanted with human cells and/or expressing human gene products recapitulate the human evolutionary specialization and diversity of genotypic and phenotypic traits

  • Very promising models are human immune system (HIS) mice generated with a mouse strain expressing several human cytokines such as macrophage colonystimulating factors (M-CSF), interleukin 3 (IL-3), IL-6, GM-CSF, and thrombopoietin (TPO), the “MISTRG-6”, and showing improved human T, B, and natural killer (NK) cell development (Das et al, 2016; Yu et al, 2017)

  • Development of liquid and solid Patient derived xenograft (PDX) models relies on the availability of material obtained from patients with defined types of cancer, which after minimal manipulation is transferred by several routes into immunodeficient mice (Fig 1D, Table 1)

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Summary

Introduction

Mice xenotransplanted with human cells and/or expressing human gene products ( known as “humanized mice”) recapitulate the human evolutionary specialization and diversity of genotypic and phenotypic traits. *Relative human HSC engraftment and chimerism (% huCD45+ cells in mouse blood at weeks 10, 15, 20 after HCT showing gating strategies)

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