Evidence map›Paper›PMID 30381411›Full record

ArticleScience translational medicine2018

Antisense oligonucleotides targeting mutant Ataxin-7 restore visual function in a mouse model of spinocerebellar ataxia type 7.

Chenchen Niu, Thazah P Prakash, Aneeza Kim, John L Quach, Laryssa A Huryn, Yuechen Yang, Edith Lopez, Ali Jazayeri, Gene Hung, Bryce L Sopher and 4 more

Abstract read
In one paragraph

Article in Science translational medicine, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 51 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
51citing papers in PubMed, 1 pooled it
5.3field-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

51 citing papers in PubMed, 1 synthesis or guideline pooled it, 82 citations in OpenAlex.

  1. Pooled it
  2. The Versatile Applications of Antisense Oligonucleotides in Modern Medicine.International journal of molecular sciences · 2026
    Review
  3. Observational
  4. Article
  5. Article
  6. Article
  7. Review
  8. Comparison ofMolecular therapy. Nucleic acids · 2025
    Article
  9. Review
  10. Review
  11. Observational
  12. Article
  13. Review
  14. Review
  15. Review
  16. Therapeutic Gene Editing in Inherited Retinal Disorders.Cold Spring Harbor perspectives in medicine · 2023
    Review
  17. Review
  18. Article
  19. Article
  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

14 authors at 5 institutions in 1 country.

Chenchen NiuDepartment of Neurology, Duke University School of Medicine, Durham, NC 27710, USA.ORCID 0000-0003-4405-9162
Thazah P PrakashIonis Pharmaceuticals, Carlsbad, CA 92008, USA.ORCID 0000-0002-7466-3312
Aneeza KimIonis Pharmaceuticals, Carlsbad, CA 92008, USA.
John L QuachDepartment of Ophthalmology, University of California, San Diego, La Jolla, CA 92093, USA.
Laryssa A HurynNational Eye Institute, National Institutes of Health, Bethesda, MD 20892, USA.
Yuechen YangDepartment of Pediatrics, University of California, San Diego, La Jolla, CA 92093, USA.ORCID 0000-0001-8893-5182
Edith LopezDepartment of Pediatrics, University of California, San Diego, La Jolla, CA 92093, USA.ORCID 0000-0002-4309-9595
Ali JazayeriIonis Pharmaceuticals, Carlsbad, CA 92008, USA.ORCID 0000-0003-4137-2292
Gene HungIonis Pharmaceuticals, Carlsbad, CA 92008, USA.ORCID 0000-0002-3840-1681
Bryce L SopherDepartment of Neurology, University of Washington, Seattle, WA 98195, USA.ORCID 0000-0001-6399-0783
Brian P BrooksNational Eye Institute, National Institutes of Health, Bethesda, MD 20892, USA.ORCID 0000-0002-1916-7551
Eric E SwayzeIonis Pharmaceuticals, Carlsbad, CA 92008, USA.ORCID 0000-0001-6430-6576
C Frank BennettIonis Pharmaceuticals, Carlsbad, CA 92008, USA.ORCID 0000-0001-9887-6251
Albert R La SpadaDepartment of Neurology, Duke University School of Medicine, Durham, NC 27710, USA. al.laspada@duke.edu.ORCID 0000-0001-6151-2964
Ionis Pharmaceuticals (United States) · USUniversity of California San Diego · USDuke University · USNational Institutes of Health · USUniversity of Washington · US

Funding

Vision BiostatisticsP30EY022589 · NEI · UNIVERSITY OF CALIFORNIA, SAN DIEGO · PI Derek Stuart Welsbie · 2012 to 2026
$10.3M
SCA7 RETINAL DEGENERATION: MOLECULAR BASIS &THERAPYR01EY014061 · NEI · UNIVERSITY OF WASHINGTON · PI ALBERT R LA SPADA · 2002 to 2026
$8.4M
Natural History of Spinocerebellar Ataxia Type 7 (SCA7)ZIAEY000553 · NEI · NATIONAL EYE INSTITUTE · PI HURYN, LARYSSA · 2016 to 2025
$2.7M
Ataxin-7 oligonucleotide knock-down to treat SCA7 retinal and cerebellar diseaseR01EY024747 · NEI · UNIVERSITY OF CALIFORNIA, SAN DIEGO · PI LA SPADA, ALBERT R · 2014 to 2017
$2.0M
NEI NIH HHS P30 EY022589NEI NIH HHS R01 EY014061NEI NIH HHS R01 EY024747
6 · The paper itself

Abstract

Spinocerebellar ataxia type 7 (SCA7) is an autosomal dominant neurodegenerative disorder characterized by cerebellar and retinal degeneration, and is caused by a CAG-polyglutamine repeat expansion in the

Indexed as

AnimalsAtaxin-7Chromatin Assembly and DisassemblyDisease Models, AnimalDisease ProgressionEpigenesis, GeneticGene Expression RegulationHumansIntravitreal InjectionsMiceMutant ProteinsOligonucleotides, AntisensePeptidesPhenotypePhotoreceptor Cells, VertebrateProtein AggregatesAtaxin-7Mutant ProteinsOligonucleotides, AntisensePeptidespolyglutamineProtein Aggregates

Identifiers

PMID30381411
PMCPMC6411060
OpenAlexW2898632421

What OpenQuestion holds

Textmetadata
LicenceTDM
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.