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Humanized Models For Disease Research And Treatment Insights

Genome Edited Animal Models For Studying Your Gene Of Interest

“When people talk about ‘model organisms,’ many people think that the animal has to ‘model’ the human disease condition to have any impact. We argue that this is not the case. Simply studying what the homologous gene does in the context of the humanized zebrafish model organism can provide important insights into disease pathology.

Humanized animal models, including humanized models for disease research and treatment insights, play a crucial role in understanding genetic mutations and disease biology discovery. “Rare genetic variants can be modeled in a number of different model organisms, informing causality between a particular variant and a pathogenic phenotype and offering clinicians and their patients hope for a diagnosis, insight into the mechanisms and pathways involved in a rare disease, and ways it might be treated.” Neff, E.P. Model matchmaking. Lab Anim 50, 39–42 (2021).


Why Choose Our C. elegans Custom Injection Service | InVivo Biosystems

Using CRISPR and other genome editing methods, InVivo Biosystems can humanize or genetically engineer zebrafish and C. elegans into powerful humanized animal models for better understanding genetic mutations and disease biology discovery.

“InVivo Biosystems adapted to the needs of our project and created strains that introduced the variants of interest at the endogenous C.elegans locus.” In doing so, “they acted as a temporary post-doc, to speed this project along much faster than we could have done in the lab ourselves.” Ellen Gregory, PhD candidate, Starr & Luxton Labs, UC Davis.

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Humanized Models For Disease Research and Treatment Insights

1. What are Humanized Models in Disease Research?

Humanized models are innovative tools in biomedical research that involve modifying animal models, such as mice or C. elegans, to carry human genes, human cells, or human immune system components. These models are pivotal for studying complex human responses to diseases and treatments in a controlled environment. By incorporating human donor genetic material or humanized immune system mice (e.g., HIS-NSG mice), researchers can observe the effects of diseases and treatments with greater relevance to human physiology, leading to more accurate predictions of clinical outcomes in drug discovery and gene therapy.

2. How do Humanized Models Improve Drug Discovery Processes?

Humanized models significantly enhance the drug discovery process by providing a more accurate representation of how therapeutic agents interact with human biology. These models, including humanized mouse models and patient-derived xenografts (PDX), allow for the evaluation of drug efficacy and toxicity in a system that closely mimics human disease states. Utilizing vivo models and preclinical models enables the identification of potential adverse reactions and cytokine release syndrome risks before clinical trials, optimizing drug screening protocols and improving the safety and effectiveness of new treatments.

3. What Role Do Humanized Models Play in Understanding Infectious and Autoimmune Diseases?

Humanized models play a crucial role in understanding infectious diseases and autoimmune diseases by simulating the human immune response within an animal model. Through the introduction of human immune cells or immune system components into immunodeficient mouse models, researchers can study the host immune interactions, immune cell chimerism, and the development of diseases within a human-like immune environment. These insights are essential for developing targeted therapies that can modulate the immune response, offering hope for patients with conditions previously considered untreatable.

4. How are Humanized Models Developed for Disease Research?

The development of humanized models for disease research involves several sophisticated techniques, including CRISPR/Cas9 for genome editing and the engraftment of human cells or tissues into immunodeficient mice. Whole gene humanization swaps animal genes for their human counterparts, while point mutation humanization introduces specific clinical variants into the animal genome. This precise genetic engineering, exemplified by the creation of humanized mouse models like HIL-NSG mice and humanized immune system mice, allows for the detailed study of human gene function, immune cell subsets, and disease mechanisms.

5. What are the Challenges and Limitations of Using Humanized Models in Research?

Despite their significant advantages, humanized models also face challenges and limitations. The complexity of the human immune system and the intricate interplay of immune cell subsets may not be fully replicated in animal models. Long-term studies and weeks post-engraftment observations are required to understand the immune cell composition and myeloid cell reconstitution dynamics. Moreover, ethical considerations and the need for human donor tissues can limit the availability of certain models. Researchers must also navigate the genetic modifications and natural killer cell activities that can differ between the model and human conditions, potentially affecting the accuracy of study results.

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