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Abstract
RNAi technology has brought a new category of treatments for various diseases including
genetic diseases, viral diseases, and cancer. Despite the great versatility of RNAi
that can down regulate almost any protein in the cells, the delicate and precise machinery
used for silencing is the same. The major challenge indeed for RNAi-based therapy
is the delivery system. In this review, we start with the uniqueness and mechanism
of RNAi machinery and the utility of RNAi in therapeutics. Then we discuss the challenges
in systemic siRNA delivery by dividing them into two categories-kinetic and physical
barriers. At the end, we discuss different strategies to overcome these barriers,
especially focusing on the step of endosome escape. Toxicity issues and current successful
examples for lipid-based delivery are also included in the review.