Evidence map›Paper›PMID 35017301›Full record

ArticleProceedings of the National Academy of Sciences of the United States of America2022

Exon-skipping antisense oligonucleotides for cystic fibrosis therapy.

Young Jin Kim, Nicole Sivetz, Jessica Layne, Dillon M Voss, Lucia Yang, Qian Zhang, Adrian R Krainer

Open access · hybridAbstract read
In one paragraph

Article in Proceedings of the National Academy of Sciences of the United States of America, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 30 papers.

0numbers the graph read from it
0cells of the map it votes in
30citing papers in PubMed
5.5field-weighted citation impact, top 3% of its field
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

30 citing papers in PubMed, 38 citations in OpenAlex.

  1. Article
  2. Article
  3. Review
  4. Review
  5. Review
  6. Expansion of Splice-Switching Therapy with Antisense Oligonucleotides.International journal of molecular sciences · 2025
    Review
  7. Article
  8. WhenFrontiers in pharmacology · 2025
    Article
  9. Article
  10. Systematic deletion of symmetricalNAR molecular medicine · 2024
    Article
  11. Article
  12. Article
  13. Article
  14. Networking to OptimizeBiomedicines · 2023
    Article
  15. Review
  16. Review
  17. Article
  18. Use of 2,6-diaminopurine as a potent suppressor of UGA premature stop codons in cystic fibrosis.Molecular therapy : the journal of the American Society of Gene Therapy · 2023
    Article
  19. Review
  20. Review
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

7 authors at 1 institution in 1 country.

Young Jin KimCold Spring Harbor Laboratory, Cold Spring Harbor, NY 11724.ORCID 0000-0003-1876-3359
Nicole SivetzCold Spring Harbor Laboratory, Cold Spring Harbor, NY 11724.ORCID 0000-0002-0825-3622
Jessica LayneCold Spring Harbor Laboratory, Cold Spring Harbor, NY 11724.ORCID 0000-0003-3371-6796
Dillon M VossCold Spring Harbor Laboratory, Cold Spring Harbor, NY 11724.ORCID 0000-0001-7481-9455
Lucia YangCold Spring Harbor Laboratory, Cold Spring Harbor, NY 11724.ORCID 0000-0002-7881-9724
Qian ZhangCold Spring Harbor Laboratory, Cold Spring Harbor, NY 11724.ORCID 0000-0001-6721-3594
Adrian R KrainerCold Spring Harbor Laboratory, Cold Spring Harbor, NY 11724; krainer@cshl.edu.ORCID 0000-0001-9024-9501
Cold Spring Harbor Laboratory · US

Funding

Single-Cell Biology Shared ResourceP30CA045508 · NCI · COLD SPRING HARBOR LABORATORY · PI David A Tuveson · 1987 to 2026
$118.9M
MEDICAL SCIENTIST TRAINING PROGRAMT32GM007367 · NIGMS · COLUMBIA UNIV NEW YORK MORNINGSIDE · PI REINER, STEVEN L · 1985 to 2021
$25.1M
MEDICAL SCIENTIST TRAINING PROGRAMT32GM008444 · NIGMS · STATE UNIVERSITY NEW YORK STONY BROOK · PI FROHMAN, MICHAEL A. · 1992 to 2024
$12.6M
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
Antisense-oligonucleotide-directed inhibition of nonsense-mediated mRNA decay of CFTR geneF30HL137326 · NHLBI · STATE UNIVERSITY NEW YORK STONY BROOK · PI KIM, YOUNG JIN · 2018 to 2021
$171k
NCI NIH HHS P30 CA045508NHLBI NIH HHS F30 HL137326NIGMS NIH HHS R01 GM042699NIGMS NIH HHS R37 GM042699NIGMS NIH HHS T32 GM007367NIGMS NIH HHS T32 GM008444
6 · The paper itself

Abstract

Mutations in the cystic fibrosis transmembrane conductance regulator (

Indexed as

Genetic TherapyCystic FibrosisCystic Fibrosis Transmembrane Conductance RegulatorExonsHEK293 CellsHumansOligonucleotides, AntisenseRNA, MessengerRNA Splice SitesCFTR protein, humanCystic Fibrosis Transmembrane Conductance RegulatorOligonucleotides, AntisenseRNA, MessengerRNA Splice Sitesantisense oligonucleotidecystic fibrosisexon skippingmRNA splicingnonsense mutation

Identifiers

PMID35017301
PMCPMC8784140
OpenAlexW4225308694

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.