Possible Therapeutic Applications and Challenges
Given the ability to knock down, in essence, any gene of interest, RNAi via siRNAs has generated a great deal of interest in both basic and applied biology. There are an increasing number of large-scale RNAi screens that are designed to identify the important genes in various biological pathways. Because disease processes also depend on the activity of multiple genes, it is expected that in some situations turning off the activity of a gene with an siRNA could produce a therapeutic benefit.
However, applying RNAi via siRNAs to living animals, especially humans, poses many challenges. Under experiments, siRNAs show different effectiveness in different cell types in a manner as yet poorly understood: Some cells respond well to siRNAs and show a robust knockdown, whereas others show no such knockdown (even despite efficient transfection).
Phase I results of the first two therapeutic RNAi trials (indicated for age-related macular degeneration, aka AMD) reported at the end of 2005 that siRNAs are well tolerated and have suitable pharmacokinetic properties.
Proof of concept trials have indicated that Ebola-targeted siRNAs may be effective as post-exposure prophylaxis in humans, with 100% of non-human primates surviving a lethal dose of Zaire Ebolavirus, the most lethal strain.
Read more about this topic: Small Interfering RNA
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