Biodistribution of phosphodiester and phosphorothioate siRNA.
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.
Departments of Pharmacology and Biochemistry, University of Texas Southwestern Medical Center, Dallas, TX 75390-9041, USA.
Dwaine A
DA
Braasch
Zain
Z
Paroo
Anca
A
Constantinescu
Gang
G
Ren
Orhan K
OK
Oz
Ralph P
RP
Mason
David R
DR
Corey
D000818:Animals; D001682:Biological Availability; D001921:Brain / Q000378:metabolism; D007668:Kidney / Q000378:metabolism; D008099:Liver / Q000378:metabolism; D051379:Mice; D009841:Oligonucleotides / Q000235:genetics / Q000493:pharmacokinetics; D009928:Organ Specificity; D010755:Organophosphates / Q000493:pharmacokinetics; D034741:RNA, Small Interfering / Q000235:genetics / Q000493:pharmacokinetics*; D014018:Tissue Distribution
D009841:Oligonucleotides; D010755:Organophosphates; D034741:RNA, Small Interfering