Evidence map›Paper›PMID 36935427›Full record

ArticleGene therapy2023

Gene augmentation for autosomal dominant retinitis pigmentosa using rhodopsin genomic loci nanoparticles in the P23H

Simna Sp, Rajendra N Mitra, Min Zheng, Jared D Chrispell, Kai Wang, Yong-Su Kwon, Ellen R Weiss, Zongchao Han

Abstract read
PubMed Publisher
In one paragraph

Article in Gene therapy, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing papers in PubMed
0.9field-weighted citation impact, top 25% 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

4 citing papers in PubMed, 6 citations in OpenAlex.

  1. Article
  2. Article
  3. Review
  4. Nanotechnology in Retinal Disease: Current Concepts and Future Directions.Journal of ocular pharmacology and therapeutics : the official journal of the Association for Ocular Pharmacology and Therapeutics
    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

8 authors at 1 institution in 1 country.

Simna Sp *Department of Ophthalmology, the University of North Carolina at Chapel Hill, Chapel Hill, NC, 27599, USA.
Rajendra N Mitra *Department of Ophthalmology, the University of North Carolina at Chapel Hill, Chapel Hill, NC, 27599, USA.
Min ZhengDepartment of Ophthalmology, the University of North Carolina at Chapel Hill, Chapel Hill, NC, 27599, USA.
Jared D ChrispellDepartment of Cell Biology and Physiology, the University of North Carolina, Chapel Hill, NC, 27599, USA.
Kai WangDepartment of Ophthalmology, the University of North Carolina at Chapel Hill, Chapel Hill, NC, 27599, USA.
Yong-Su KwonDepartment of Ophthalmology, the University of North Carolina at Chapel Hill, Chapel Hill, NC, 27599, USA.
Ellen R WeissDepartment of Cell Biology and Physiology, the University of North Carolina, Chapel Hill, NC, 27599, USA.
Zongchao HanDepartment of Ophthalmology, the University of North Carolina at Chapel Hill, Chapel Hill, NC, 27599, USA. zongchao@med.unc.edu.ORCID 0000-0002-2019-395X
University of North Carolina at Chapel Hill · US

Funding

DESENSITIZATION OF CONE VISUAL SIGNALING PATHWAYSR01EY012224 · NEI · UNIV OF NORTH CAROLINA CHAPEL HILL · PI WEISS, ELLEN RUTH · 2000 to 2019
$5.4M
Targeting Retinitis Pigmentosa Using Nanoparticle-Mediated Delivery of Genomic DNAR01EY026564 · NEI · UNIV OF NORTH CAROLINA CHAPEL HILL · PI HAN, ZONGCHAO · 2016 to 2020
$1.9M
Identification of novel contributors to retinitis pigmentosa using metabolic and proteomic approachesR01EY032089 · NEI · UNIV OF NORTH CAROLINA CHAPEL HILL · PI WEISS, ELLEN RUTH · 2021 to 2023
$1.7M
NEI NIH HHS R01 EY012224NEI NIH HHS R01 EY026564NEI NIH HHS R01 EY032089
6 · The paper itself

Abstract

Gene therapy for autosomal dominant retinitis pigmentosa (adRP) is challenged by the dominant inheritance of the mutant genes, which would seemingly require a combination of mutant suppression and wild-type replacement of the appropriate gene. We explore the possibility that delivery of a nanoparticle (NP)-mediated full-length mouse genomic rhodopsin (gRho) or human genomic rhodopsin (gRHO) locus can overcome the dominant negative effects of the mutant rhodopsin in the clinically relevant P23H

Indexed as

MicroRNAsNanoparticlesRetinitis PigmentosaAnimalsDisease Models, AnimalGenomicsHumansMiceMutationRhodopsinMicroRNAsRhodopsin

Identifiers

PMID36935427
OpenAlexW4327900128

What OpenQuestion holds

Textmetadata
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.