Department of Veterinary Sciences; and Laboratory for Functional Genome Analysis (LAFUGA), Gene Center, LMU Munich, Feodor-Lynen-Str. 25, 81377 Munich, Germany.
J Mol Med (Berl). 2010 Jul;88(7):653-64. doi: 10.1007/s00109-010-0610-9. Epub 2010 Mar 26.
The translation of novel discoveries from basic research to clinical application is a long, often inefficient, and thus costly process. Accordingly, the process of drug development requires optimization both for economic and for ethical reasons, in order to provide patients with appropriate treatments in a reasonable time frame. Consequently, "Translational Medicine" became a top priority in national and international roadmaps of human health research. Appropriate animal models for the evaluation of efficacy and safety of new drugs or therapeutic concepts are critical for the success of translational research. In this context rodent models are most widely used. At present, transgenic pigs are increasingly being established as large animal models for selected human diseases. The first pig whole genome sequence and many other genomic resources will be available in the near future. Importantly, efficient and precise techniques for the genetic modification of pigs have been established, facilitating the generation of tailored disease models. This article provides an overview of the current techniques for genetic modification of pigs and the transgenic pig models established for neurodegenerative diseases, cardiovascular diseases, cystic fibrosis, and diabetes mellitus.
将新发现从基础研究转化为临床应用是一个漫长的过程，往往效率低下，因此成本高昂。因此，出于经济和伦理原因，药物开发过程需要进行优化，以便在合理的时间内为患者提供适当的治疗。因此，“转化医学”成为国家和国际人类健康研究路线图上的首要任务。对于新药物或治疗概念的疗效和安全性评估，合适的动物模型对于转化研究的成功至关重要。在这方面，啮齿动物模型应用最为广泛。目前，转基因猪越来越多地被建立为特定人类疾病的大动物模型。第一个猪全基因组序列和许多其他基因组资源将在不久的将来提供。重要的是，已经建立了用于猪基因修饰的高效和精确技术，促进了定制疾病模型的产生。本文概述了目前用于猪基因修饰的技术以及为神经退行性疾病、心血管疾病、囊性纤维化和糖尿病建立的转基因猪模型。
