Treating Disease at the RNA Level with Oligonucleotides

As research on the structure and function of RNAs has rapidly expanded, so too has the number of oligonucleotide drugs in clinical development that regulate RNA. This article describes several examples and suggests where the field is headed.

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Bibliographic Details
Published inThe New England journal of medicine Vol. 380; no. 1; pp. 57 - 70
Main Author Levin, Arthur A
Format Journal Article
LanguageEnglish
Published United States Massachusetts Medical Society 03.01.2019
SeriesFrontiers in Medicine
Subjects
Clinical trials
Deoxyribonucleic acid
Disease
DNA
Drug development
Drugs
Enzymes
FDA approval
Federal regulation
Gene expression
Gene Expression - drug effects
Genomes
Humans
MicroRNAs
Morpholinos - therapeutic use
Muscular Atrophy, Spinal - genetics
Muscular Atrophy, Spinal - therapy
Muscular dystrophy
Muscular Dystrophy, Duchenne - genetics
Muscular Dystrophy, Duchenne - therapy
Mutation
Oligonucleotides
Oligonucleotides - pharmacology
Oligonucleotides - therapeutic use
Proteins
Ribonucleic acid
RNA
RNA, Messenger - metabolism
RNA, Small Interfering - therapeutic use
Structure-function relationships
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More Information
Summary:As research on the structure and function of RNAs has rapidly expanded, so too has the number of oligonucleotide drugs in clinical development that regulate RNA. This article describes several examples and suggests where the field is headed.
Bibliography:ObjectType-Article-2
SourceType-Scholarly Journals-1
ObjectType-Feature-3
content type line 23
ObjectType-Review-1
ISSN:0028-4793
1533-4406
DOI:10.1056/NEJMra1705346