Walsh Nicole C, Kenney Laurie L, Jangalwe Sonal, Aryee Ken-Edwin, Greiner Dale L, Brehm Michael A, Shultz Leonard D
Department of Molecular Medicine, Diabetes Center of Excellence, University of Massachusetts Medical School, Worcester, Massachusetts 01605.
The Jackson Laboratory, Bar Harbor, Maine 04609; email:
Annu Rev Pathol. 2017 Jan 24;12:187-215. doi: 10.1146/annurev-pathol-052016-100332. Epub 2016 Dec 5.
Immunodeficient mice engrafted with functional human cells and tissues, that is, humanized mice, have become increasingly important as small, preclinical animal models for the study of human diseases. Since the description of immunodeficient mice bearing mutations in the IL2 receptor common gamma chain (IL2rg) in the early 2000s, investigators have been able to engraft murine recipients with human hematopoietic stem cells that develop into functional human immune systems. These mice can also be engrafted with human tissues such as islets, liver, skin, and most solid and hematologic cancers. Humanized mice are permitting significant progress in studies of human infectious disease, cancer, regenerative medicine, graft-versus-host disease, allergies, and immunity. Ultimately, use of humanized mice may lead to the implementation of truly personalized medicine in the clinic. This review discusses recent progress in the development and use of humanized mice and highlights their utility for the study of human diseases.
移植了功能性人类细胞和组织的免疫缺陷小鼠，即人源化小鼠，作为用于研究人类疾病的小型临床前动物模型，正变得越来越重要。自21世纪初描述了白细胞介素2受体共同γ链（IL2rg）发生突变的免疫缺陷小鼠以来，研究人员已能够将小鼠受体移植入可发育成功能性人类免疫系统的人类造血干细胞。这些小鼠还可以移植人类组织，如胰岛、肝脏、皮肤以及大多数实体癌和血液癌。人源化小鼠在人类传染病、癌症、再生医学、移植物抗宿主病、过敏和免疫研究中取得了重大进展。最终，使用人源化小鼠可能会导致临床上真正实现个性化医疗。本文综述了人源化小鼠开发和使用方面的最新进展，并强调了它们在人类疾病研究中的效用。
