Pharmacology of Antisense Drugs

Recent studies have led to a greater appreciation of the diverse roles RNAs play in maintaining normal cellular function and how they contribute to disease pathology, broadening the number of potential therapeutic targets. Antisense oligonucleotides are the most direct means to target RNA in a selec...

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Vydáno v:Annual review of pharmacology and toxicology Ročník 57; s. 81 - 105
Hlavní autoři: Bennett, C Frank, Baker, Brenda F, Pham, Nguyen, Swayze, Eric, Geary, Richard S
Médium: Journal Article
Jazyk:angličtina
Vydáno: United States 06.01.2017
Témata:
Animals
Autoimmune Diseases - drug therapy
Autoimmune Diseases - metabolism
Cardiovascular Diseases - drug therapy
Cardiovascular Diseases - metabolism
Humans
Oligonucleotides, Antisense - metabolism
Oligonucleotides, Antisense - pharmacology
Oligonucleotides, Antisense - therapeutic use
RNA Interference - drug effects
RNA Interference - physiology
RNA Splicing - drug effects
RNA Splicing - physiology
RNA interference
RNA
eteplirsen
antisense oligonucleotides
RNase H
nusinersen
patisiran
mipomersen
RNA splicing
oligonucleotide
ISSN:1545-4304
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Popis
Shrnutí:Recent studies have led to a greater appreciation of the diverse roles RNAs play in maintaining normal cellular function and how they contribute to disease pathology, broadening the number of potential therapeutic targets. Antisense oligonucleotides are the most direct means to target RNA in a selective manner and have become an established platform technology for drug discovery. There are multiple molecular mechanisms by which antisense oligonucleotides can be used to modulate RNAs in cells, including promoting the degradation of the targeted RNA or modulating RNA function without degradation. Antisense drugs utilizing various antisense mechanisms are demonstrating therapeutic potential for the treatment of a broad variety of diseases. This review focuses on some of the advances that have taken place in translating antisense technology from the bench to the clinic.
Bibliografie:ObjectType-Article-2
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ObjectType-Review-1
ISSN:1545-4304
DOI:10.1146/annurev-pharmtox-010716-104846