Opportunities and challenges in modeling human brain disorders in transgenic primates
Opportunities and challenges in modeling human brain disorders in transgenic primates
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DOI：
10.1038/nn.4362
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发表时间：
2016-09-01
影响因子：
25
通讯作者：
Feng, Guoping
中科院分区：
文献类型：
--
作者：
Jennings, Charles;Landman, Rogier;Feng, Guoping
分子遗传学工具对神经科学产生了深远影响，但直到最近，它们的应用在很大程度上局限于少数模式物种，最显著的是小鼠、斑马鱼、黑腹果蝇和秀丽隐杆线虫。随着CRISPR等新的基因组工程技术的发展，在更广泛的物种（包括非人灵长类动物）中应用这些分子工具变得越来越可行。这将为大脑研究带来许多机遇，但也会带来挑战。在此，我们根据近期和可预见的技术进步，指出其中的一些机遇和挑战，并提出一些建议。我们主要关注创建新的灵长类疾病模型，以了解脑部疾病的病理机制，并开发有效治疗的新方法。然而，我们也强调，灵长类遗传模型在解决许多关于大脑功能的基本问题方面具有巨大潜力，为疾病研究的未来进展提供了重要基础。
Molecular genetic tools have had a profound impact on neuroscience, but until recently their application has largely been confined to a few model species, most notably mouse, zebrafish, Drosophila melanogaster and Caenorhabditis elegans. With the development of new genome engineering technologies such as CRISPR, it is becoming increasingly feasible to apply these molecular tools in a wider range of species, including nonhuman primates. This will lead to many opportunities for brain research, but it will also pose challenges. Here we identify some of these opportunities and challenges in light of recent and foreseeable technological advances and offer some suggestions. Our main focus is on the creation of new primate disease models for understanding the pathological mechanisms of brain disorders and for developing new approaches to effective treatment. However, we also emphasize that primate genetic models have great potential to address many fundamental questions about brain function, providing an essential foundation for future progress in disease research.