Proteins are considered to be the key players in
structure, function, and metabolic regulation of our bodies.
The mechanisms used in conventional therapies often
rely on inhibition of proteins with small molecules, but
another promising method to treat disease is by targeting
the corresponding mRNAs. In 1998, Craig Mellow and
Andrew Fire discovered dsRNA-mediated gene silencing
via RNA interference or RNAi. This discovery introduced
almost unlimited possibilities for new gene silencing
methods, thus opening new doors to clinical medicine.
RNAi is a biological process that inhibits gene expression
by targeting the mRNA. RNAi-based therapeutics have
several potential advantages (i) a priori ability to target
any gene, (ii) relatively simple design process, (iii) sitespecificity,
(iv) potency, and (v) a potentially safe and
selective knockdown of the targeted cells. However,
the problem lies within the formulation and delivery of
RNAi therapeutics including rapid excretion, instability in the bloodstream, poor cellular uptake, and inefficient
intracellular release. In an attempt to solve these issues,
different types of RNAi therapeutic delivery strategies
including multifunctional RNA nanoparticles are being
developed. In this mini-review, we will briefly describe
some of the current approaches.