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Molecules 2016, 21(9), 1249; doi:10.3390/molecules21091249

Delivery of RNAi Therapeutics to the Airways—From Bench to Bedside

Department of Pharmacology and Pharmacy, Li Ka Shing Faculty of Medicine, The University of Hong Kong, 21 Sassoon Road, Pokfulam, Hong Kong, China
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Author to whom correspondence should be addressed.
Academic Editor: Wong Wai-Shiu
Received: 16 August 2016 / Revised: 5 September 2016 / Accepted: 13 September 2016 / Published: 20 September 2016
(This article belongs to the Special Issue Nucleic Acid-based Drug)
View Full-Text   |   Download PDF [1057 KB, uploaded 20 September 2016]   |  

Abstract

RNA interference (RNAi) is a potent and specific post-transcriptional gene silencing process. Since its discovery, tremendous efforts have been made to translate RNAi technology into therapeutic applications for the treatment of different human diseases including respiratory diseases, by manipulating the expression of disease-associated gene(s). Similar to other nucleic acid-based therapeutics, the major hurdle of RNAi therapy is delivery. Pulmonary delivery is a promising approach of delivering RNAi therapeutics directly to the airways for treating local conditions and minimizing systemic side effects. It is a non-invasive route of administration that is generally well accepted by patients. However, pulmonary drug delivery is a challenge as the lungs pose a series of anatomical, physiological and immunological barriers to drug delivery. Understanding these barriers is essential for the development an effective RNA delivery system. In this review, the different barriers to pulmonary drug delivery are introduced. The potential of RNAi molecules as new class of therapeutics, and the latest preclinical and clinical studies of using RNAi therapeutics in different respiratory conditions are discussed in details. We hope this review can provide some useful insights for moving inhaled RNAi therapeutics from bench to bedside. View Full-Text
Keywords: RNA interference; short interfering RNA (siRNA); short hairpin RNA (shRNA); microRNA (miRNA); pulmonary delivery; respiratory diseases; inhalation RNA interference; short interfering RNA (siRNA); short hairpin RNA (shRNA); microRNA (miRNA); pulmonary delivery; respiratory diseases; inhalation
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This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. (CC BY 4.0).

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MDPI and ACS Style

Qiu, Y.; Lam, J.K.W.; Leung, S.W.S.; Liang, W. Delivery of RNAi Therapeutics to the Airways—From Bench to Bedside. Molecules 2016, 21, 1249.

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