Evidence map›Paper›PMID 27893103›Full record

ArticleInvestigative ophthalmology & visual science2016

Potential of Small Molecule-Mediated Reprogramming of Rod Photoreceptors to Treat Retinitis Pigmentosa.

Paul A Nakamura, Shibing Tang, Andy A Shimchuk, Sheng Ding, Thomas A Reh

Open access · goldAbstract read
In one paragraph

Article in Investigative ophthalmology & visual science, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers.

0numbers the graph read from it
0cells of the map it votes in
18citing papers in PubMed
1.4field-weighted citation impact, top 17% 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

18 citing papers in PubMed, 26 citations in OpenAlex.

  1. Review
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  5. Germline knockout ofProceedings of the National Academy of Sciences of the United States of America · 2024
    Article
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  7. Article
  8. Review
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  11. Rhodopsin as a Molecular Target to Mitigate Retinitis Pigmentosa.Advances in experimental medicine and biology · 2022
    Review
  12. Article
  13. Review
  14. Review
  15. Review
  16. Article
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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

5 authors at 2 institutions in 1 country.

Paul A NakamuraDepartment of Biological Structure, University of Washington, School of Medicine, Seattle, Washington, United States.
Shibing TangUniversity of California-San Francisco, UCSF School of Pharmacy, Department of Pharmaceutical Chemistry, San Francisco California, United States.
Andy A ShimchukDepartment of Biological Structure, University of Washington, School of Medicine, Seattle, Washington, United States.
Sheng DingUniversity of California-San Francisco, UCSF School of Pharmacy, Department of Pharmaceutical Chemistry, San Francisco California, United States.
Thomas A RehDepartment of Biological Structure, University of Washington, School of Medicine, Seattle, Washington, United States.
University of Washington · USUniversity of California, San Francisco · US

Funding

Vision Research CoreP30EY001730 · NEI · UNIVERSITY OF WASHINGTON · PI MAUREEN E NEITZ · 1985 to 2026
$23.5M
Regeneration of Retinal Neurons by Chemically Induced Reprogramming of Muller GliR01EY021374 · NEI · UNIVERSITY OF CALIFORNIA, SAN DIEGO · PI DING, SHENG, REH, THOMAS A · 2010 to 2014
$4.2M
NEI NIH HHS P30 EY001730NEI NIH HHS R01 EY021374
6 · The paper itself

Abstract

Purpose: Mutations in rod photoreceptor genes can cause retinitis pigmentosa (RP). Rod gene expression is regulated by the nuclear hormone receptor, Nr2e3. Genetic deletion of Nr2e3 reprograms rods into cells that resemble cone photoreceptors, and might therefore prevent their death from some forms of RP. There are no identified ligands for Nr2e3; however, reverse agonists might mimic the genetic rescue effect and may be therapeutically useful for the treatment of RP. Methods: We screened for small molecule modulators of Nr2e3 using primary retinal cell cultures and characterized the most potent, which we have named photoregulin1 (PR1), in vitro and in vivo. We also tested the ability of PR1 to slow the progression of photoreceptor degeneration in two common mouse models of autosomal dominant RP, the RhoP23H and the Pde6brd1 mutations. Results: In developing retina, PR1 causes a decrease in rod gene expression and an increase in S opsin+ cones. Photoregulin1 continues to inhibit rod gene expression in adult mice. When applied to two mouse models of RP, PR1 slows the degeneration of photoreceptors. Conclusions: Chemical compounds identified as modulators of Nr2e3 activity may be useful for the treatment of RP through their effects on expression of disease-causing mutant genes.

Indexed as

Gene Expression Regulation, DevelopmentalAnimalsAnimals, NewbornBlotting, WesternCells, CulturedCyclic Nucleotide Phosphodiesterases, Type 6Disease Models, AnimalDNADNA Mutational AnalysisIn Situ Nick-End LabelingMiceMice, Inbred C57BLMutationOrphan Nuclear ReceptorsReal-Time Polymerase Chain ReactionRetinaCyclic Nucleotide Phosphodiesterases, Type 6DNANr2e3 protein, mouseOrphan Nuclear ReceptorsPde6b protein, mouse

Identifiers

PMID27893103
PMCPMC5134355
OpenAlexW2557845633

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.