Vandamme Thierry F
University of Strasbourg, Faculty of Pharmacy, UMR 7199 CNRS, Laboratory of Concept and Application of Bioactive Molecules, Biogalenic Team, 74 Route du Rhin, 67400 Illkirch Graffenstaden, France.
J Pharm Bioallied Sci. 2014 Jan;6(1):2-9. doi: 10.4103/0975-7406.124301.
Advances in molecular biology have significantly increased the understanding of the biology of different diseases. However, these discoveries have not yet been fully translated into improved treatments for patients with diseases such as cancers. One of the factors limiting the translation of knowledge from preclinical studies to the clinic has been the limitations of in vivo diseases models. In this brief review, we will discuss the advantages and disadvantages of rodent models that have been developed to simulate human pathologies, focusing in models that employ xenografts and genetic modification. Within the framework of genetically engineered mouse (GEM) models, we will review some of the current genetic strategies for modeling diseases in the mouse and the preclinical studies that have already been undertaken. We will also discuss how recent improvements in imaging technologies may increase the information derived from using these GEMs during early assessments of potential therapeutic pathways. Furthermore, it is interesting to note that one of the values of using a mouse model is the very rapid turnover rate of the animal, going through the process of birth to death in a very short timeframe relative to that of larger mammalian species.
分子生物学的进展显著增进了人们对不同疾病生物学特性的理解。然而，这些发现尚未完全转化为针对癌症等疾病患者的改进治疗方法。限制知识从临床前研究转化到临床的因素之一是体内疾病模型的局限性。在这篇简短的综述中，我们将讨论为模拟人类病理状况而开发的啮齿动物模型的优缺点，重点关注采用异种移植和基因改造的模型。在基因工程小鼠（GEM）模型的框架内，我们将回顾一些目前在小鼠中模拟疾病的遗传策略以及已经开展的临床前研究。我们还将讨论成像技术最近的改进如何在潜在治疗途径的早期评估中增加使用这些GEM所获得的信息。此外，值得注意的是，使用小鼠模型的价值之一在于动物的更替速度非常快，相对于大型哺乳动物物种而言，在很短的时间内经历从出生到死亡的过程。
