Evidence map›Paper›PMID 21472006›Full record

ArticleGene therapy2011

Selective atonal gene delivery improves balance function in a mouse model of vestibular disease.

C Schlecker, M Praetorius, D E Brough, R G Presler, C Hsu, P K Plinkert, H Staecker

Open access · greenAbstract read
In one paragraph

Article in Gene therapy, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 45 papers.

0numbers the graph read from it
0cells of the map it votes in
45citing papers in PubMed
3.4field-weighted citation impact, top 7% 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

45 citing papers in PubMed, 83 citations in OpenAlex.

  1. Trial
  2. Article
  3. Article
  4. Advancing precision ear medicine: leveraging animal models for disease insights and therapeutic innovations.Mammalian genome : official journal of the International Mammalian Genome Society · 2025
    Review
  5. Review
  6. Article
  7. Review
  8. Article
  9. Third-generation lentiviral gene therapy rescues function in a mouse model of Usher 1B.Molecular therapy : the journal of the American Society of Gene Therapy · 2023
    Article
  10. Article
  11. Article
  12. Repair of surviving hair cells in the damaged mouse utricle.Proceedings of the National Academy of Sciences of the United States of America · 2022
    Article
  13. Article
  14. Review
  15. Regeneration of Hair Cells in the Human Vestibular System.Frontiers in molecular neuroscience · 2022
    Review
  16. Advances in genome editing for genetic hearing loss.Advanced drug delivery reviews · 2021
    Review
  17. Quantitative Assessment of Anti-Gravity Reflexes to Evaluate Vestibular Dysfunction in Rats.Journal of the Association for Research in Otolaryngology : JARO · 2019
    Article
  18. Review
  19. Article
  20. Article
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 3 institutions in 2 countries.

C SchleckerDepartment of Otolaryngology, Head and Neck Surgery, University of Kansas School of Medicine, Kansas city, KS, USA.
M Praetorius
D E Brough
R G Presler
C Hsu
P K Plinkert
H Staecker
GenVec · USUniversity of Kansas · USHeidelberg University · DE

Funding

Research Design and Analysis Core (RDAC)P30HD002528 · NICHD · UNIVERSITY OF KANSAS LAWRENCE · PI FOWLER, STEPHEN CLETIUS · 1985 to 2015
$22.1M
Development of atonal therapy for bilateral vestibular hypofunctionR01DC008424 · NIDCD · UNIVERSITY OF KANSAS MEDICAL CENTER · PI STAECKER, HINRICH · 2007 to 2011
$2.0M
NICHD NIH HHS HD02528NICHD NIH HHS P30 HD002528NIDCD NIH HHS R01 DC008424NIDCD NIH HHS R01DC008424
6 · The paper itself

Abstract

Loss of balance is often due to loss of vestibular hair cells. In mammals, regeneration of functional hair cells in the mature sensory epithelium is limited; therefore, loss of sensory cells can lead to debilitating balance problems. Delivery of the transcription factor atonal (atoh1) after aminoglycoside ototoxicity has previously been shown to induce the transdifferentiation of supporting cells into new hair cells and restore function. A problem with mouse aminoglycoside models is that the partial loss of hair cells seen in human disease is difficult to establish consistently. To more closely mirror human clinical balance dysfunction, we have used systemic application of 3,3'-iminodipropionitrile (IDPN), a vestibulotoxic nitrile compound known to cause vestibular hair cell loss, to induce a consistent partial loss of vestibular hair cells. To determine if balance function could be restored, we delivered atoh1 using a new adenovirus vector, based on Ad28. The Ad28 adenovector is based on a human serotype with a low seroprevalence that appears to target gene delivery to vestibular supporting cells. To further provide cell type selectivity of gene delivery, we expressed atoh1 using the supporting cell-specific glial fibrillary acid protein promoter. Delivery of this vector to IDPN-damaged vestibular organs resulted in a significant recovery of vestibular hair cells and restoration of balance, as measured by time on rotarod compared with untreated controls.

Indexed as

Gene Transfer TechniquesAdenoviridaeAnimalsBasic Helix-Loop-Helix ProteinsDisease Models, AnimalFemaleGenetic TherapyGenetic VectorsGlial Fibrillary Acidic ProteinHair Cells, VestibularHumansMiceMice, Inbred C57BLNitrilesPostural BalanceRegeneration3,3'-iminodipropionitrileATOH1 protein, humanBasic Helix-Loop-Helix ProteinsGlial Fibrillary Acidic ProteinNitriles

Identifiers

PMID21472006
PMCPMC3136627
OpenAlexW1965873234

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.