Chemistry, structure and function of approved oligonucleotide therapeutics

Abstract Eighteen nucleic acid therapeutics have been approved for treatment of various diseases in the last 25 years. Their modes of action include antisense oligonucleotides (ASOs), splice-switching oligonucleotides (SSOs), RNA interference (RNAi) and an RNA aptamer against a protein. Among the di...

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Vydané v:	Nucleic acids research Ročník 51; číslo 6; s. 2529 - 2573
Hlavní autori:	Egli, Martin, Manoharan, Muthiah
Médium:	Journal Article
Jazyk:	English
Vydavateľské údaje:	England Oxford University Press 11.04.2023
Predmet:	Critical Reviews and s Humans Morpholinos - pharmacology Muscular Dystrophy, Duchenne - drug therapy Muscular Dystrophy, Duchenne - genetics Oligonucleotides, Antisense - chemistry Oligonucleotides, Antisense - metabolism Oligonucleotides, Antisense - therapeutic use RNA - chemistry RNA Interference
ISSN:	0305-1048, 1362-4962, 1362-4962
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