摘要
小干扰RNA（siRNA）是一种序列特异性的、在转录后水平调控基因表达的小RNA（small RNA）分子。实验研究证实，siRNA可有效用于治疗骨质疏松、内分泌肿瘤以及糖代谢异常、肥胖、糖尿病慢性并发症、脂代谢异常等代谢性疾病。siRNA药物治疗内分泌疾病的临床研究目前主要集中于代谢性疾病，如siRNA药物bevasiranib已进入治疗糖尿病视网膜病变（黄斑水肿）的Ⅱ期临床试验。
Small interfering RNA（siRNA） is a small RNA molecule which regulates gene expression in post-transcriptional level by sequence-specific mechanism. Studies show that siRNA can be applied in treating osteoporosis, endocrine tumors and metabolic diseases such as glyeometabolism abnormalities, obesity, diabetic chronic complications, and lipid metabolism abnormalities. The clinical trials of siRNA drug used in endocrine diseases are mainly focused on metabolic diseases. For example, bevasiranib-a siRNA drug used to treat diabetic retinopathy（maenlar edema） has been in phase Ⅱ clinical trial.
出处
《国际内分泌代谢杂志》
2008年第1期26-29,共4页
International Journal of Endocrinology and Metabolism
基金
国家自然科学基金资助项目（30770854）
