Agah R, Frenkel P A, French B A, Michael L H, Overbeek P A, Schneider M D
Molecular Cardiology Unit, Baylor College of Medicine, Houston, Texas 77030, USA.
J Clin Invest. 1997 Jul 1;100(1):169-79. doi: 10.1172/JCI119509.
Mouse models of human disease can be generated by homologous recombination for germline loss-of-function mutations. However, embryonic-lethal phenotypes and systemic, indirect dysfunction can confound the use of knock-outs to elucidate adult pathophysiology. Site-specific recombination using Cre recombinase can circumvent these pitfalls, in principle, enabling temporal and spatial control of gene recombination. However, direct evidence is lacking for the feasibility of Cre-mediated recombination in postmitotic cells. Here, we exploited transgenic mouse technology plus adenoviral gene transfer to achieve Cre-mediated recombination in cardiac muscle. In vitro, Cre driven by cardiac-specific alpha-myosin heavy chain (alphaMyHC) sequences elicited recombination selectively at loxP sites in purified cardiac myocytes, but not cardiac fibroblasts. In vivo, this alphaMyHC-Cre transgene elicited recombination in cardiac muscle, but not other organs, as ascertained by PCR analysis and localization of a recombination-dependent reporter protein. Adenoviral delivery of Cre in vivo provoked recombination in postmitotic, adult ventricular myocytes. Recombination between loxP sites was not detected in the absence of Cre. These studies demonstrate the feasibility of using Cre-mediated recombination to regulate gene expression in myocardium, with efficient induction of recombination even in terminally differentiated, postmitotic muscle cells. Moreover, delivery of Cre by viral infection provides a simple strategy to control the timing of recombination in myocardium.
人类疾病的小鼠模型可通过同源重组产生种系功能丧失突变。然而,胚胎致死表型以及全身性、间接功能障碍可能会干扰使用基因敲除来阐明成体病理生理学。原则上,使用Cre重组酶的位点特异性重组可以规避这些缺陷,从而实现基因重组的时空控制。然而,缺乏关于有丝分裂后细胞中Cre介导的重组可行性的直接证据。在这里,我们利用转基因小鼠技术和腺病毒基因转移在心肌中实现Cre介导的重组。在体外,由心脏特异性α-肌球蛋白重链(αMyHC)序列驱动的Cre在纯化的心肌细胞而非心脏成纤维细胞的loxP位点选择性地引发重组。在体内,通过PCR分析和重组依赖性报告蛋白的定位确定,这种αMyHC-Cre转基因在心肌而非其他器官中引发重组。体内腺病毒递送Cre可在有丝分裂后的成年心室肌细胞中引发重组。在没有Cre的情况下未检测到loxP位点之间的重组。这些研究证明了使用Cre介导的重组来调节心肌中基因表达的可行性,即使在终末分化的有丝分裂后肌肉细胞中也能有效诱导重组。此外,通过病毒感染递送Cre提供了一种控制心肌中重组时间的简单策略。