The recently identified 2-component clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 system has revolutionized the ease with which genome editing can be performed, greatly simplifying the process of generating knockout animal models.1,2 However, in spite of the great advances made, fewer studies have focused on the efficacy of the system for editing postnatal or adult tissues as a means to model adult onset diseases or to provide potential therapeutic intervention. In particular, the use of the system to perform genome editing in a tissue-specific manner remains to be thoroughly evaluated, especially for tissues that are less amenable to viral delivery of the CRISPR/Cas9 components, a common strategy for delivery in adult tissues.3 Because the heart is responsible for a large disease burden, it represents a particularly attractive tissue to target for therapeutic purposes.4 Although several studies have shown that cardiomyocytes can be edited in the postnatal murine heart using viral delivery of CRISPR/Cas9 system components,5–7 the efficiency of this phenomenon has been less well characterized. Article, see p 1168 A study in this issue by Johansen et al8 from Eva van Rooij’s group performed an in-depth analysis of CRISPR/Cas9-mediated gene editing in postnatal mouse myocytes. Cardiac-restricted expression of Cas9 was inducing using mice containing a floxed Cas9 cassette with a GFP (green fluorescent protein) reporter knocked into the Rosa26 locus together with an αMHC-Cre (alpha-myosin heavy chain-Cre) transgene. Consistent with other models that have expressed Cas9 in either a broad or tissue restricted manner,5,6,9 no changes in baseline cardiac function were observed after Cas9 overexpression, suggesting that Cas9 …

中文翻译：

---

心脏CRISPR的注意事项

最近鉴定出的2组分成簇的规则间隔的短回文重复序列（CRISPR）/ Cas9系统彻底改变了基因组编辑的简便性，大大简化了基因敲除动物模型的生成过程1,2。取得了进步，很少有研究集中在用于编辑产后或成人组织的系统的功效上，该系统可作为模拟成人发病疾病或提供潜在治疗干预的手段。特别地，使用该系统以组织特异性方式进行基因组编辑仍有待彻底评估，尤其是对于不适合于病毒递送CRISPR / Cas9成分的组织，这是在成人组织中递送的常见策略。 3因为心脏是疾病的重担，它代表了一种特别有吸引力的组织，可用于治疗。4尽管一些研究表明，使用CRISPR / Cas9系统组件的病毒递送可以在产后鼠心中编辑心肌细胞，[5-7]这种现象的效率较差。表征。文章，见第1168页。Evan van Rooij的研究小组的Johansen等[8]对此研究进行了深入分析，分析了CRISPR / Cas9介导的产后小鼠心肌细胞中的基因编辑。使用含有带有GFP（绿色荧光蛋白）报告基因和αMHC-Cre（α-肌球蛋白重链-Cre）转基因敲入GFP（绿色荧光蛋白）报告基因的Cas9盒的小鼠，诱导了Cas9的心脏限制性表达。与其他以广泛或组织受限的方式表达Cas9的模型一致[5,6，