Abstract
Oligonucleotides (oligos) have been under clinical development for approximately the past 30 years, beginning with antisense oligonucleotides (ASOs) and apatmers and followed about 15 years ago by siRNAs. During that lengthy period of time, numerous clinical trials have been performed and thousands of trial participants accrued onto studies. Of all the molecules evaluated as of January 2017, the regulatory authorities assessed that six provided clear clinical benefit in rigorously controlled trials. The story of these six is given in this review.
Keywords:
Defitelio; Eteplirsen; Kynamro; Macugen; Spinraza; Vitravene; aptamer; oligonucleotide; phosphorothioate; therapeutics.
Copyright © 2017 The American Society of Gene and Cell Therapy. Published by Elsevier Inc. All rights reserved.
MeSH terms
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Aptamers, Nucleotide / therapeutic use*
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Clinical Trials as Topic
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Cytomegalovirus Retinitis / genetics
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Cytomegalovirus Retinitis / therapy
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Cytomegalovirus Retinitis / virology
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Drug Approval
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Hepatic Veno-Occlusive Disease / genetics
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Hepatic Veno-Occlusive Disease / pathology
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Hepatic Veno-Occlusive Disease / therapy
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Humans
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Hypercholesterolemia / genetics
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Hypercholesterolemia / pathology
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Hypercholesterolemia / therapy
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Macular Degeneration / genetics
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Macular Degeneration / pathology
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Macular Degeneration / therapy
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Morpholinos / therapeutic use*
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Muscular Atrophy, Spinal / genetics
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Muscular Atrophy, Spinal / pathology
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Muscular Atrophy, Spinal / therapy
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Muscular Dystrophy, Duchenne / genetics
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Muscular Dystrophy, Duchenne / pathology
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Muscular Dystrophy, Duchenne / therapy
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Oligonucleotides / therapeutic use*
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Oligonucleotides, Antisense / therapeutic use*
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Polydeoxyribonucleotides / therapeutic use*
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Thionucleotides / therapeutic use*
Substances
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Aptamers, Nucleotide
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Morpholinos
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Oligonucleotides
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Oligonucleotides, Antisense
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Polydeoxyribonucleotides
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Thionucleotides
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pegaptanib
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defibrotide
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nusinersen
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mipomersen
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eteplirsen
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fomivirsen