Evidence map›Paper›PMID 22262036›Full record

ReviewNature reviews. Drug discovery2012

RNA therapeutics: beyond RNA interference and antisense oligonucleotides.

Ryszard Kole, Adrian R Krainer, Sidney Altman

Abstract readReview
In one paragraph

Review in Nature reviews. Drug discovery, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 578 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
578citing papers in PubMed, 1 pooled it
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

578 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Article
  3. Review
  4. PCBP2 facilitates miR-93-5p-mediated repression ofMolecular therapy. Nucleic acids · 2026
    Article
  5. Article
  6. Review
  7. Article
  8. Article
  9. RNA-Based Therapies for Inherited Metabolic Disorders.Journal of inherited metabolic disease · 2026
    Review
  10. Review
  11. Review
  12. CPP-PNA Conjugate-Mediated Inhibition ofInternational journal of molecular sciences · 2026
    Article
  13. Review
  14. Challenges and Perspectives in ncRNA Research.Experientia supplementum (2012) · 2026
    Review
  15. Target, silence, replace: a review on RNA-based drugs in modern medicine.Frontiers in cell and developmental biology · 2026
    Review
  16. Article
  17. Review
  18. Article
  19. Review
  20. Tissue Pharmacokinetics of Antisense Oligonucleotides.Methods in molecular biology (Clifton, N.J.) · 2026
    Article

518 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

3 authors.

Ryszard KoleAVI BioPharma, 3450 Monte Villa Parkway, Bothell, Washington 98021, USA. rkole@avibio.com
Adrian R Krainer
Sidney Altman

Funding

VIRAL TRANSACTIVATIONP01CA013106 · NCI · COLD SPRING HARBOR LABORATORY · PI William Richard McCombie · 1985 to 2026
$116.8M
BIOCHEMISTRY OF PREMRNA SPLICINGR01GM042699 · NIGMS · COLD SPRING HARBOR LABORATORY · PI Adrian R Krainer · 1989 to 2026
$8.8M
Biochemistry of Pre-mRNA SplicingR37GM042699 · NIGMS · COLD SPRING HARBOR LABORATORY · PI KRAINER, ADRIAN R · 2012 to 2021
$7.7M
NCI NIH HHS P01 CA013106NIGMS NIH HHS R01 GM042699NIGMS NIH HHS R37 GM042699
6 · The paper itself

Abstract

Here, we discuss three RNA-based therapeutic technologies exploiting various oligonucleotides that bind to RNA by base pairing in a sequence-specific manner yet have different mechanisms of action and effects. RNA interference and antisense oligonucleotides downregulate gene expression by inducing enzyme-dependent degradation of targeted mRNA. Steric-blocking oligonucleotides block the access of cellular machinery to pre-mRNA and mRNA without degrading the RNA. Through this mechanism, steric-blocking oligonucleotides can redirect alternative splicing, repair defective RNA, restore protein production or downregulate gene expression. Moreover, they can be extensively chemically modified to acquire more drug-like properties. The ability of RNA-blocking oligonucleotides to restore gene function makes them best suited for the treatment of genetic disorders. Positive results from clinical trials for the treatment of Duchenne muscular dystrophy show that this technology is close to achieving its clinical potential.

Indexed as

RNA InterferenceAlternative SplicingAnimalsAnti-Bacterial AgentsAntiviral Agentsbeta-ThalassemiaGene ExpressionHumansMorpholinosMuscular Dystrophy, DuchenneMyotonic DystrophyOligodeoxyribonucleotides, AntisensePhosphorothioate OligonucleotidesRNA, MessengerRNA, Small InterferingAnti-Bacterial AgentsAntiviral AgentsMorpholinosOligodeoxyribonucleotides, AntisensePhosphorothioate OligonucleotidesRNA, MessengerRNA, Small Interfering

Identifiers

PMID22262036
PMCPMC4743652

What OpenQuestion holds

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Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.