id	author	title	date	pages	extension	mime	words	sentences	flesch	summary	cache	txt
cord-337199-mbv8kd1k	Ballarín-González, Borja	Clinical translation of RNAi-based treatments for respiratory diseases	2012-09-07		.txt	text/plain	10541	571	41	This review outlines the essential steps required for the clinical translation of RNAi-based respiratory therapies including disease and RNA target selection, siRNA design, respiratory barriers, and delivery solutions. 1 In vitro screening and selection of siRNA candidates against potential targets such as pathogenic or host factors; 2 optimize siRNA designs with high silencing activity and avoid immune recognition, increase stability, and reduce off-target effects; 3 evaluation of cytokine profile and gene silencing efficiency in preclinical disease models using either naked or a suitable delivery system; and 4 clinical trials to evaluate toxicity and biological activity of lead formulations Considerable efforts have attempted to harness the "exogenous" or "endogenous" branches of the RNAi pathway for therapeutic purposes. The authors suggested that the observed therapeutic effect reported in early studies was most likely due to activation of the innate immune response by the recognition of viral-specific nonmodified siRNA sequences by toll-like receptors (TLRs) instead of an RNAi-mediated effect.	./cache/cord-337199-mbv8kd1k.txt	./txt/cord-337199-mbv8kd1k.txt