Braasch Dwaine A, Paroo Zain, Constantinescu Anca, Ren Gang, Oz Orhan K, Mason Ralph P, Corey David R
Departments of Pharmacology and Biochemistry, University of Texas Southwestern Medical Center, Dallas, TX 75390-9041, USA.
Bioorg Med Chem Lett. 2004 Mar 8;14(5):1139-43. doi: 10.1016/j.bmcl.2003.12.074.
Short interfering RNAs (siRNAs) are valuable tools for analyzing protein function in mammalian cell culture. This success has led to high expectations for in vivo and therapeutic applications. However, the pharmacokinetic properties of siRNA are not known. Here we report the biodistribution of a phosphodiester (PO) siRNA duplex and examine the effect of phosphorothioate (PS) linkages. Our findings indicate that biodistribution of siRNA is similar to that for single-stranded antisense oligonucleotides and offer insights for use of siRNA in vivo.
小干扰RNA（siRNA）是分析哺乳动物细胞培养中蛋白质功能的宝贵工具。这一成功使得人们对其体内和治疗应用寄予厚望。然而，siRNA的药代动力学特性尚不清楚。在此，我们报告了一种磷酸二酯（PO）siRNA双链体的生物分布，并研究了硫代磷酸酯（PS）连接的作用。我们的研究结果表明，siRNA的生物分布与单链反义寡核苷酸相似，并为siRNA在体内的应用提供了见解。
