Naomi Berrie Diabetes Center and Division of Molecular Genetics, Department of Pediatrics, Columbia University, New York, New York, United States of America.
Department of Biostatistics, Mailman School of Public Health, Columbia University, New York, New York, United States of America.
PLoS One. 2018 May 30;13(5):e0197548. doi: 10.1371/journal.pone.0197548. eCollection 2018.
We have previously reported that Ildr2 knockdown via adenovirally-delivered shRNA causes hepatic steatosis in mice. In the present study we investigated hepatic biochemical and anatomic phenotypes of Cre-mediated Ildr2 knock-out mice. Liver-specific Ildr2 knock-out mice were generated in C57BL/6J mice segregating for a floxed (exon 1) allele of Ildr2, using congenital and acute (10-13-week-old male mice) Cre expression. In addition, Ildr2 shRNA was administered to Ildr2 knock-out mice to test the effects of Ildr2 shRNA, per se, in the absence of Ildr2 expression. RNA sequencing was performed on livers of these knockdown and knockout mice. Congenital and acute liver-specific and hepatocyte-specific knockout mice did not develop hepatic steatosis. However, administration of Ildr2 shRNA to Ildr2 knock-out mice did cause hepatic steatosis, indicating that the Ildr2 shRNA had apparent "off-target" effects on gene(s) other than Ildr2. RNA sequencing and BLAST sequence alignment revealed Dgka as a candidate gene mediating these "off-target" effects. Ildr2 shRNA is 63% homologous to the Dgka gene, and Dgka expression decreased only in mice displaying hepatic steatosis. Dgka encodes diacylglycerol kinase (DGK) alpha, one of a family of DGKs which convert diacylglycerides to phosphatidic acid for second messenger signaling. Dgka knockdown mice would be expected to accumulate diacylglyceride, contributing to the observed hepatic steatosis. We conclude that ILDR2 plays a negligible role in hepatic steatosis. Rather, hepatic steatosis observed previously in Ildr2 knockdown mice was likely due to shRNA targeting of Dgka and/or other "off-target" genes. We propose that the gene candidates identified in this follow-up study may lead to identification of novel regulators of hepatic lipid metabolism.
我们之前报道过,腺病毒介导的 shRNA 敲低 Ildr2 会导致小鼠肝脂肪变性。在本研究中,我们研究了 Cre 介导的 Ildr2 敲除小鼠的肝生化和解剖表型。利用 Ildr2 的 floxed(外显子 1)等位基因,在 C57BL/6J 小鼠中生成了肝特异性 Ildr2 敲除小鼠,通过先天性和急性(10-13 周龄雄性小鼠) Cre 表达。此外,还向 Ildr2 敲除小鼠中给予了 Ildr2 shRNA,以测试在没有 Ildr2 表达的情况下 Ildr2 shRNA 本身的作用。对这些敲低和敲除小鼠的肝脏进行了 RNA 测序。先天性和急性肝特异性和肝细胞特异性敲除小鼠未发生肝脂肪变性。然而,向 Ildr2 敲除小鼠中给予 Ildr2 shRNA 确实导致了肝脂肪变性,表明 Ildr2 shRNA 对 Ildr2 以外的基因具有明显的“脱靶”作用。RNA 测序和 BLAST 序列比对显示 Dgka 是介导这些“脱靶”作用的候选基因。Ildr2 shRNA 与 Dgka 基因有 63%的同源性,并且仅在显示肝脂肪变性的小鼠中 Dgka 的表达降低。Dgka 编码二酰基甘油激酶(DGK)alpha,是 DGK 家族的一员,该家族将二酰基甘油转化为磷脂酸以进行第二信使信号转导。预计 Dgka 敲低小鼠会积累二酰基甘油,导致观察到的肝脂肪变性。我们得出结论,ILDR2 在肝脂肪变性中作用微不足道。相反,以前在 Ildr2 敲低小鼠中观察到的肝脂肪变性可能是由于 shRNA 靶向 Dgka 和/或其他“脱靶”基因。我们提出,在这项后续研究中鉴定的候选基因可能会导致鉴定新的肝脂质代谢调节剂。