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Comparison of Genetically Engineered Immunodeficient Animal Models for Nonclinical Testing of Stem Cell Therapies
Pharmaceutics ( IF 4.9 ) Pub Date : 2021-01-20 , DOI: 10.3390/pharmaceutics13020130
Yoon-Young Kim , Jin-Soo Kim , Jeong-Hwan Che , Seung-Yup Ku , Byeong-Cheol Kang , Jun-Won Yun

For the recovery or replacement of dysfunctional cells and tissue—the goal of stem cell research—successful engraftment of transplanted cells and tissues are essential events. The event is largely dependent on the immune rejection of the recipient; therefore, the immunogenic evaluation of candidate cells or tissues in immunodeficient animals is important. Understanding the immunodeficient system can provide insights into the generation and use of immunodeficient animal models, presenting a unique system to explore the capabilities of the innate immune system. In this review, we summarize various immunodeficient animal model systems with different target genes as valuable tools for biomedical research. There have been numerous immunodeficient models developed by different gene defects, resulting in many different features in phenotype. More important, mice, rats, and other large animals exhibit very different immunological and physiological features in tissue and organs, including genetic background and a representation of human disease conditions. Therefore, the findings from this review may guide researchers to select the most appropriate immunodeficient strain, target gene, and animal species based on the research type, mutant gene effects, and similarity to human immunological features for stem cell research.

中文翻译:

基因工程免疫缺陷动物模型对干细胞疗法非临床测试的比较

对于功能失调的细胞和组织的恢复或替换(干细胞研究的目标),成功植入移植的细胞和组织是至关重要的事件。该事件很大程度上取决于受体的免疫排斥。因此,免疫缺陷动物中候选细胞或组织的免疫原性评估很重要。了解免疫缺陷系统可以提供有关免疫缺陷动物模型的产生和使用的见解,从而提供一个独特的系统来探索先天免疫系统的功能。在这篇综述中,我们总结了具有不同靶基因的各种免疫缺陷动物模型系统,作为生物医学研究的重要工具。由不同的基因缺陷发展了许多免疫缺陷模型,导致表型有许多不同的特征。更重要的是,小鼠,大鼠和其他大型动物在组织和器官中表现出非常不同的免疫和生理特征,包括遗传背景和人类疾病状况的表征。因此,本综述的发现可能会指导研究人员根据研究类型,突变基因的作用以及与人类干细胞研究的免疫学特征的相似性,选择最合适的免疫缺陷株,靶基因和动物物种。
更新日期:2021-01-20
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