Clinical potential of oligonucleotide-based therapeutics in the respiratory system

Moschos, Sterghios, Usher, Louise and Lindsay, Mark (2017) Clinical potential of oligonucleotide-based therapeutics in the respiratory system. Pharmacology & Therapeutics, 169. pp. 83-103. ISSN 0163-7258

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The discovery of an ever-expanding plethora of coding and non-coding RNAs with nodal and causal roles in the regulation of lung physiology and disease is reinvigorating interest in the clinical utility of the oligonucleotide therapeutic class. This is strongly supported through recent advances in nucleic acids chemistry, synthetic oligonucleotide delivery and viral gene therapy that have succeeded in bringing to market at least three nucleic acid-based drugs. As a consequence, multiple new candidates such as RNA interference modulators, antisense, and splice switching compounds are now progressing through clinical evaluation. Here, manipulation of RNA for the treatment of lung disease is explored, with emphasis on robust pharmacological evidence aligned to the five pillars of drug development: exposure to the appropriate tissue, binding to the desired molecular target, evidence of the expected mode of action, activity in the relevant patient population and commercially viable value proposition.

Item Type: Article
Uncontrolled Keywords: Oligonucleotide therapeutics, siRNA, miRNA, PNA, PPMO, delivery
Subjects: A100 Pre-clinical Medicine
B200 Pharmacology, Toxicology and Pharmacy
C100 Biology
C700 Molecular Biology, Biophysics and Biochemistry
G300 Statistics
Department: Faculties > Health and Life Sciences > Applied Sciences
Depositing User: Sterghios Moschos
Date Deposited: 25 Oct 2016 08:26
Last Modified: 01 Aug 2021 05:38

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