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Mouse Model Strategies & Allele Types

Humanized Mouse Models

A genetically engineered mouse in which one or more human genes, immune system components, or biological pathways have been introduced to replicate aspects of human physiology, bridging the gap between basic research and clinical translation.

Overview

Traditional mouse models provide powerful insights into gene function and disease mechanisms—but their biology can differ significantly from humans. Humanized mouse models solve this challenge by integrating human genetic material or biological components directly into the mouse genome or immune system.

Types of Humanized Models

Gene replacement models replace mouse genes with human orthologs. Immune system humanization integrates human immune components. Cell and tissue engraftment models use human hematopoietic stem cells or tumor tissues in immunodeficient mice.

Frequently Asked Questions

What's the difference between a knockin and a humanized mouse model?

A knockin mouse model introduces a new sequence into a specific genomic locus. A humanized mouse model specifically incorporates human genes, proteins, or immune components to replicate human biology.

Are humanized mice suitable for preclinical drug testing?

Yes. Humanized mice are commonly used for preclinical testing because they express human-specific targets and pathways, enabling accurate evaluation of biologics, gene therapies, and targeted drugs.

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Related Terms

Knockin (KI) Mouse Models

A genetically engineered mouse in which a specific DNA sequence—such as a gene, cDNA, mutation, reporter, or human ortholog—is inserted into a defined genomic locus to add or modify gene function.

Humanized Immune Checkpoint (PD-1 / PD-L1 / CTLA-4)

Genetically engineered mice with key inhibitory immune receptors—such as PD-1, PD-L1, and CTLA-4—replaced with their human equivalents. Essential for evaluating antibody and biologic therapies targeting immune checkpoint pathways.

Cytokine / Receptor Humanization

The genetic replacement of mouse cytokines, receptors, or ligand–receptor pairs with their human equivalents. These models recreate critical components of the human immune network for accurate evaluation of human biologics.

More in Mouse Model Strategies & Allele Types

Knockout (KO) Mouse ModelsConditional Knockout (cKO) Mouse ModelsConventional (Constitutive) Knockout Mouse ModelsKnockin (KI) Mouse ModelsReporter Gene / Reporter Allele
Humanized Immune Checkpoint (PD-1 / PD-L1 / CTLA-4)View All TermsHypomorphic / Null Allele

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