[EN] KETOHEXOKINASE (KHK) IRNA COMPOSITIONS AND METHODS OF USE THEREOF<br/>[FR] COMPOSITIONS D'ARNI DE CÉTOHEXOKINASE (KHK) ET PROCÉDÉS POUR LES UTILISER
申请人:ALNYLAM PHARMACEUTICALS INC
公开号:WO2015123264A1
公开(公告)日:2015-08-20
The present invention relates to RNAi agents, e.g., double- stranded RNAi agents, targeting the ketohexokinase (KHK) gene, and methods of using such RNAi agents to inhibit expression of KHK and methods of treating subjects having a KHK-associated disorder, e.g., liver disease (e.g., fatty liver, steatohepatitis), dyslipidemia (e.g., hyperlipidemia, high LDL cholesterol, low HDL cholesterol, hypertriglyceridemia, postprandial hypertriglyceridemia), disorders of glycemic control (e.g., insulin resistance, diabetes), cardiovascular disease (e.g., hypertension, endothelial cell dysfunction), kidney disease (e.g., acute kidney disorder, tubular dysfunction, proinflammatory changes to the proximal tubules), metabolic syndrome, adipocyte dysfunction, visceral adipose deposition, obesity, hyperuricemia, gout, eating disorders, and excessive sugar craving.
本发明涉及RNAi剂,例如,靶向酮
己糖激酶(KHK)
基因的双链RNAi剂,以及使用这种RNAi剂抑制KHK表达的方法,以及治疗患有KHK相关疾病的受试者的方法,例如肝病(例如脂肪肝,脂肪肝炎),血脂异常(例如高血脂,高LDL
胆固醇,低HDL
胆固醇,高三酸
甘油酯血症,餐后高三酸
甘油酯血症),糖代谢控制紊乱(例如
胰岛素抵抗,糖尿病),心血管疾病(例如高血压,内皮细胞功能障碍),肾脏疾病(例如急性肾脏疾病,肾小管功能障碍,近曲小管的前炎性变化),代谢综合征,脂肪细胞功能障碍,内脏脂肪沉积,肥胖,高
尿酸血症,痛风,进食障碍和过度食糖欲望。