Transcriptomic profile of Pcy mice treated with RGLS4326

Kidney global mRNA expression profiles was carried out in the Pcy/CD1 model following RGLS4326 treatment versus PBS-treated controls. We found that RGLS4326 treatment had a significant transcriptome-wide impact and globally de-repressed mRNAs of predicted miR-17 target genes in cystic kidneys of Pcy/CD1 model 5-weeks old mice were randomly assigned and dosed with RGLS4326 (25 mg kg-1) or PBS by SC injections once- monthly (Q4W) for 25 weeks. All animals were euthanized, and kidney harvested at 30 weeks of age. Five untreated male WT/CD1 mice were included for comparison.

本研究针对Pcy/CD1模型小鼠开展肾脏全局信使RNA（messenger RNA, mRNA）表达谱分析，对比RGLS4326治疗组与磷酸盐缓冲液（phosphate buffered saline, PBS）对照组的检测结果。实验将5周龄的Pcy/CD1模型小鼠随机分组，通过皮下（subcutaneous, SC）注射给予RGLS4326（25 mg·kg⁻¹）或PBS，每月给药1次（每4周一次，Q4W），持续给药25周；所有小鼠均在30周龄时实施安乐死并采集肾脏组织。另纳入5只未接受处理的雄性野生型WT/CD1小鼠作为对照。研究结果显示，RGLS4326治疗可产生显著的全转录组（transcriptome）调控效应，并在该模型小鼠的囊性肾脏中，全局性解除了预测为miR-17（microRNA-17）靶基因的mRNA的转录抑制状态。