SiRNA or salt and medicine for inhibiting TMPRSS6 gene expression and application of siRNA or salt and medicine

The present invention provides siRNA or a pharmaceutically acceptable salt for inhibiting the expression of TMPRSS6 in human cells, and the siRNA is suitably modified so as to improve the silence ability of a target and reduce non-target activity. The invention also provides a biological preparation or a medicinal preparation which contains the siRNA and is used for inhibiting the expression of the TMPRSS6. Through a series of in-vitro and in-vivo tests, the siRNA sequence with very good biological activity for inhibiting TMPRSS6 expression is obtained, the activity of the siRNA sequence is higher than that of TMPRSS6-HCM-9 which clinically progresses fastest, and the siRNA sequence is expected to be clinically applied to treatment of diseases related to TMPRSS6 expression abnormity or iron overload or iron overload related diseases and has very good application prospects. 本发明提供了用于抑制人体细胞中TMPRSS6表达的siRNA或者药学上可接受的盐，以及对所述siRNA进行了合适的修饰，以提高靶标的沉默能力并降低非靶点活性。本发明还提供了含有上述siRNA的用于抑制TMPRSS6表达的生物制剂或者药物制剂。本发明通过一系列体外和体内试验中，