Department of Molecular Biology, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.
Proc Natl Acad Sci U S A. 2013 Apr 2;110(14):5588-93. doi: 10.1073/pnas.1301019110. Epub 2013 Mar 4.
Reprogramming of mouse fibroblasts toward a myocardial cell fate by forced expression of cardiac transcription factors or microRNAs has recently been demonstrated. The potential clinical applicability of these findings is based on the minimal regenerative potential of the adult human heart and the limited availability of human heart tissue. An initial but mandatory step toward clinical application of this approach is to establish conditions for conversion of adult human fibroblasts to a cardiac phenotype. Toward this goal, we sought to determine the optimal combination of factors necessary and sufficient for direct myocardial reprogramming of human fibroblasts. Here we show that four human cardiac transcription factors, including GATA binding protein 4, Hand2, T-box5, and myocardin, and two microRNAs, miR-1 and miR-133, activated cardiac marker expression in neonatal and adult human fibroblasts. After maintenance in culture for 4-11 wk, human fibroblasts reprogrammed with these proteins and microRNAs displayed sarcomere-like structures and calcium transients, and a small subset of such cells exhibited spontaneous contractility. These phenotypic changes were accompanied by expression of a broad range of cardiac genes and suppression of nonmyocyte genes. These findings indicate that human fibroblasts can be reprogrammed to cardiac-like myocytes by forced expression of cardiac transcription factors with muscle-specific microRNAs and represent a step toward possible therapeutic application of this reprogramming approach.
通过强制表达心脏转录因子或 microRNAs 使鼠成纤维细胞向心肌细胞命运重编程最近已经得到证实。这些发现的潜在临床应用基于成人心脏的最小再生潜能和人类心脏组织的有限可用性。将这种方法应用于临床的初始但强制性步骤是建立将成人成纤维细胞转化为心脏表型的条件。为此,我们试图确定直接重编程人类成纤维细胞为心肌细胞所需的最佳因素组合。在这里,我们表明,四种人类心脏转录因子,包括 GATA 结合蛋白 4、Hand2、T-box5 和肌球蛋白,以及两种 microRNAs,miR-1 和 miR-133,可激活新生儿和成纤维细胞中的心脏标志物表达。在用这些蛋白和 microRNAs 培养 4-11 周后,重编程的人类成纤维细胞显示出类似肌小节的结构和钙瞬变,并且一小部分此类细胞表现出自发收缩性。这些表型变化伴随着广泛的心脏基因表达和非心肌细胞基因的抑制。这些发现表明,通过强制表达具有肌肉特异性 microRNAs 的心脏转录因子,人类成纤维细胞可以被重编程为类似心肌的细胞,这代表了这种重编程方法可能的治疗应用的一个步骤。