RNAi as New Class of Nanomedicines - Harnessing Biomaterials in Nanomedicine: Preparation, Toxicity, and Applications - page 183

siRNA design to optimize RISC uptake and gene silencing. In spite
of these advances, there are still a number of concerns that need
to be addressed as these technologies begin to make their way into
the clinic. Foremost among these concerns is the need for a more
comprehensive understanding of the potential toxicities associated
with siRNAs and the delivery vehicles. Improvements in design and
the selective incorporation of chemical modifications have served
to reduce these off -target eff ects, but further improvements are
warranted to ensure the safety of these gene-silencing reagents.
Due to the inherent diff erences between humans and rodents, the
use of non-human primates in the drug development process may
provide a better model for understanding potential toxicities. In
addition, many of the current delivery approaches incorporate
multiple components, which increase both the complexity of their
manufacture and, as a result, the cost of their production. Although
there are still many challenges that need to be overcome, there is
hope that siRNA-based therapeutics will soon become a reality and
provide novel tools to combat a variety of diseases, particularly for
those diseases for which therapeutic options are limited or non-
existent.
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