A tiny therapeutic delivery system that can control the body’s ability to manufacture proteins has been developed by Saudi Arabia’s King Abdullah University of Science and Technology (KAUST) researchers.
Genes contain the instructions for manufacturing the proteins that make up our body. Genetic information is translated into the proteins needed to build living cells through a transcription process in which DNA’s genetic code is copied into a large molecule known as messenger RNA (mRNA).
This transcription process can be altered by introducing short double-strands of RNA, referred to as small interfering RNA (siRNA), which binds to the mRNA and inhibits the expression of particular genes. Harnessing this RNA interference for therapeutic applications is difficult and requires a material that can protect the siRNA as it travels through the bloodstream, helping it to penetrate the cell’s outer membrane and deliver it to its target location.
“Delivery of RNA is very tricky as it can be readily digested by cells. Better vehicles are needed so more RNA can be delivered in order to edit genes,” says Niveen Khashab from the KAUST Smart Hybrid Materials Laboratory.