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Journal Article
Review
Disease modeling in genetic kidney diseases: mice.
Cell and Tissue Research 2017 July
The mouse still represents arguably the most important mammal organism in research for modeling human genetic kidney diseases in vivo. Compared with many other mammal species, the breeding and maintenance of mice in the laboratory is relatively simple and cheap and reproduction cycles are short. In addition to classic gene knockout mouse lines, new molecular biological technologies have led to the development of a plethora of other, more sophisticated, mouse models, allowing the targeting of genes or gene function in a cell-specific, tissue-specific and time-dependent fashion. With the refinement of more recently developed genome-editing technologies, including the clustered regularly interspaced short palindromic repeats (CRISPR)-Cas system and other engineered nucleases such as zinc-finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs), our "tool set" of mouse models is expected to rapidly expand. These technological advances hold great promise and should enable us to study and hence understand the biology of inherited kidney diseases in greater detail. By analogy, we may be able to answer questions regarding the impact of individual proteins on the development of human kidney disorders, the underlying mechanisms governing the evolution of the disease and the predicted responsiveness to therapeutic interventions. Moreover, knockout and transgenic mouse models can be highly informative with respect to the effects of genetic variations on renal phenotypes. This review focuses on mouse models that have been devised primarily to study monogenic human kidney diseases, which are typically caused by a single abnormal gene and passed on in a Mendelian pattern. Despite the large number of human hereditary kidney disorders and the multitude of mouse models described in the literature, we attempt to give a balanced overview of several well-known renal pathologies, a few of which are addressed in some detail.
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