Clinical translation of RNAi-based treatments for respiratory diseases

Drug Delivery and Translational Research
Borja Ballarín-GonzálezKenneth Alan Howard

Abstract

The ability to harness the RNA interference (RNAi) mechanism as a potential potent therapeutic has attracted great interest from academia and industry. Numerous preclinical and recent clinical trials have demonstrated the effectiveness of RNAi triggers such as synthetic small interfering RNA (siRNA). Chemical modification and delivery technologies can be utilized to avoid immune stimulation and improve the bioactivity and pharmacokinetics. Local application to the respiratory epithelia allows direct access to the site of respiratory pathogens that include influenza and respiratory syncytial virus (RSV). 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. Attention is given to antiviral therapies and preclinical evaluation with focus on the current status of anti-RSV clinical trials.

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Citations

Jul 17, 2018·Journal of Neuromuscular Diseases·Anthony Behin, Rozen Le Panse
Dec 16, 2017·Cancer Metastasis Reviews·Xiuhui ChenAnil K Sood
Oct 11, 2020·Pharmaceuticals·Muhammad Imran SajidRakesh Kumar Tiwari
Aug 18, 2021·Acta Pharmaceutica Sinica. B·Makhloufi ZoulikhaWei He

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Methods Mentioned

BETA
chemical modifications
transgenic
bronchoalveolar lavage
chemical modification

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