RNA interference (RNAi) is a remarkable
endogenous regulatory pathway that can bring about sequence-specific gene
silencing. If harnessed effectively, RNAi could result in a potent targeted
therapeutic modality with applications ranging from viral diseases to cancer.
The major barrier to realizing the full medicinal potential of RNAi is the
difficulty of delivering effector molecules, such as small interfering RNAs
(siRNAs), in vivo. An effective delivery strategy for siRNAs must address
limitations that include poor stability and non-targeted biodistribution, while
protecting against the stimulation of an undesirable innate immune response.
The design of such a system requires rigorous understanding of all mechanisms
involved. This article reviews the mechanistic principles of RNA interference,
its potential, the greatest challenges for use in biomedical applications, and
some of the work that has been done toward engineering delivery systems that
overcome some of the hurdles facing siRNA-based therapeutics.
Source: Therapeutic siRNA: principles,
challenges, and strategies. Gavrilov K, Saltzman WM. Yale J Biol Med. 2012
Jun;85(2):187-200.
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