Meng Fantao, Han Yong, Srisai Dollada, Belakhov Valery, Farias Monica, Xu Yong, Palmiter Richard D, Baasov Timor, Wu Qi
Children's Nutrition Research Center, Department of Pediatrics, Baylor College of Medicine, Houston, TX 77030;
Department of Molecular Physiology and Biophysics, University of Iowa, Iowa City, IA 52242;
Proc Natl Acad Sci U S A. 2016 Mar 29;113(13):3645-50. doi: 10.1073/pnas.1602049113. Epub 2016 Mar 14.
Currently available inducible Cre/loxP systems, despite their considerable utility in gene manipulation, have pitfalls in certain scenarios, such as unsatisfactory recombination rates and deleterious effects on physiology and behavior. To overcome these limitations, we designed a new, inducible gene-targeting system by introducing an in-frame nonsense mutation into the coding sequence of Cre recombinase (nsCre). Mutant mRNAs transcribed from nsCre transgene can be efficiently translated into full-length, functional Cre recombinase in the presence of nonsense suppressors such as aminoglycosides. In a proof-of-concept model, GABA signaling from hypothalamic neurons expressing agouti-related peptide (AgRP) was genetically inactivated within 4 d after treatment with a synthetic aminoglycoside. Disruption of GABA synthesis in AgRP neurons in young adult mice led to a dramatic loss of body weight due to reduced food intake and elevated energy expenditure; they also manifested glucose intolerance. In contrast, older mice with genetic inactivation of GABA signaling by AgRP neurons had only transient reduction of feeding and body weight; their energy expenditure and glucose tolerance were unaffected. These results indicate that GABAergic signaling from AgRP neurons plays a key role in the control of feeding and metabolism through an age-dependent mechanism. This new genetic technique will augment current tools used to elucidate mechanisms underlying many physiological and neurological processes.
目前可用的诱导型Cre/loxP系统,尽管在基因操作中具有相当大的实用性,但在某些情况下存在缺陷,如重组率不理想以及对生理和行为产生有害影响。为了克服这些局限性,我们通过在Cre重组酶(nsCre)的编码序列中引入一个框内无义突变,设计了一种新的诱导型基因靶向系统。在存在诸如氨基糖苷类等无义抑制因子的情况下,从nsCre转基因转录的突变mRNA能够有效地翻译成全长的、有功能的Cre重组酶。在一个概念验证模型中,在用合成氨基糖苷类处理后的4天内,表达刺鼠相关肽(AgRP)的下丘脑神经元的GABA信号被基因失活。年轻成年小鼠中AgRP神经元的GABA合成中断导致由于食物摄入量减少和能量消耗增加而体重急剧下降;它们还表现出葡萄糖不耐受。相比之下,通过AgRP神经元使GABA信号基因失活的老年小鼠仅出现短暂的进食和体重下降;它们的能量消耗和葡萄糖耐受性未受影响。这些结果表明,来自AgRP神经元的GABA能信号通过一种年龄依赖性机制在进食和代谢控制中起关键作用。这种新的基因技术将增强目前用于阐明许多生理和神经过程潜在机制的工具。