Biodistribution of phosphodiester and phosphorothioate siRNA

DA Braasch, Z Paroo, A Constantinescu, G Ren… - Bioorganic & medicinal …, 2004 - Elsevier
DA Braasch, Z Paroo, A Constantinescu, G Ren, OK Öz, RP Mason, DR Corey
Bioorganic & medicinal chemistry letters, 2004Elsevier
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 …
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.
Elsevier