“RNA interference is regarded as one of the most important
therapeutic discoveries in decades,” says Dr. Guo. However, the
therapeutic relevance of small interfering RNA to silence
disease-causing genes is in jeopardy unless it can be delivered
in a targeted fashion. Davis calls pRNA “the missing link
necessary for RNA interference to be delivered in a targeted,
systemic fashion.” The most significant challenges in developing
RNA-based therapeutics have been delivery, stability, and
toxicity. To be successful, small interfering RNAs must be able
to penetrate cell membranes, resist degradation by nucleases
within cells, move into appropriate cell compartments, and home
in on specific cells. The pRNA technology meets all these
requirements without causing toxic side effects, Davis reports.
Other companies creating RNA-based therapeutics deliver the
agents to cells wrapped in liposomes or polymer structures.
These formulations are large and complex, and they reportedly
hinder the ability of the therapeutics to e