Division of Animal Science, National Swine Resource and Research Center, and.
Annu Rev Anim Biosci. 2013 Jan;1:203-19. doi: 10.1146/annurev-animal-031412-103715. Epub 2013 Jan 3.
Although pigs are used widely as models of human disease, their utility as models has been enhanced by genetic engineering. Initially, transgenes were added randomly to the genome, but with the application of homologous recombination, zinc finger nucleases, and transcription activator-like effector nuclease (TALEN) technologies, now most any genetic change that can be envisioned can be completed. To date these genetic modifications have resulted in animals that have the potential to provide new insights into human diseases for which a good animal model did not exist previously. These new animal models should provide the preclinical data for treatments that are developed for diseases such as Alzheimer's disease, cystic fibrosis, retinitis pigmentosa, spinal muscular atrophy, diabetes, and organ failure. These new models will help to uncover aspects and treatments of these diseases that were otherwise unattainable. The focus of this review is to describe genetically engineered pigs that have resulted in models of human diseases.
虽然猪被广泛用作人类疾病的模型,但通过基因工程,它们的用途得到了增强。最初,转基因是随机添加到基因组中的,但随着同源重组、锌指核酸酶和转录激活因子样效应物核酸酶(TALEN)技术的应用,现在几乎可以完成任何可以想象的遗传改变。迄今为止,这些遗传修饰已经产生了具有潜在能力的动物,可以为以前没有良好动物模型的人类疾病提供新的见解。这些新的动物模型应该为针对阿尔茨海默病、囊性纤维化、色素性视网膜炎、脊髓性肌萎缩症、糖尿病和器官衰竭等疾病开发的治疗方法提供临床前数据。这些新模型将有助于揭示这些疾病的方面和治疗方法,否则这些方面和治疗方法是无法实现的。本文的重点是描述导致人类疾病模型的基因工程猪。