Evidence map›Paper›PMID 31308478›Full record

ArticleGene therapy2019

AAVrh-10 transduces outer retinal cells in rodents and rabbits following intravitreal administration.

Yong Zeng, Haohua Qian, Zhijian Wu, Dario Marangoni, Paul A Sieving, Ronald A Bush

Open access · greenAbstract read
In one paragraph

Article in Gene therapy, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
9citing papers in PubMed, 1 pooled it
0.5field-weighted citation impact, top 35% 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

9 citing papers in PubMed, 1 synthesis or guideline pooled it, 11 citations in OpenAlex.

  1. Pooled it
  2. Review
  3. Article
  4. Review
  5. Review
  6. Rs1hGene therapy · 2022
    Article
  7. Article
  8. Article
  9. Novel AAV44.9-Based Vectors Display Exceptional Characteristics for Retinal Gene Therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2020
    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

6 authors at 1 institution in 1 country.

Yong ZengSection on Translational Research for Retinal and Macular Degeneration, National Institute on Deafness and Other Communication Disorders, National Institutes of Health, Bethesda, MD, USA.
Haohua QianNational Eye Institute, National Institutes of Health, Bethesda, MD, USA.ORCID http://orcid.org/0000-0002-1114-4697
Zhijian WuNational Eye Institute, National Institutes of Health, Bethesda, MD, USA.
Dario MarangoniSection on Translational Research for Retinal and Macular Degeneration, National Institute on Deafness and Other Communication Disorders, National Institutes of Health, Bethesda, MD, USA.
Paul A SievingSection on Translational Research for Retinal and Macular Degeneration, National Institute on Deafness and Other Communication Disorders, National Institutes of Health, Bethesda, MD, USA.ORCID http://orcid.org/0000-0002-2918-577X
Ronald A BushSection on Translational Research for Retinal and Macular Degeneration, National Institute on Deafness and Other Communication Disorders, National Institutes of Health, Bethesda, MD, USA. bushr@nidcd.nih.gov.
National Institutes of Health · US

Funding

Visual Function CoreZICEY000503 · NEI · NATIONAL EYE INSTITUTE · PI QIAN, HAOHUA · 2011 to 2025
$12.3M
Retinal Gene TherapyZIAEY000443 · NEI · NATIONAL EYE INSTITUTE · PI LI, TIANSEN · 2009 to 2020
$8.4M
Pathophysiology and Treatment of Retinal Degenerations in Animal ModelsZIADC000077 · NIDCD · NATIONAL INSTITUTE ON DEAFNESS AND OTHER COMMUNICATION DISORDERS · PI SIEVING, PAUL · 2009 to 2019
$3.2M
Evaluation of Visual Function in Animal ModelsZIAEY000530 · NEI · NATIONAL EYE INSTITUTE · PI QIAN, HAOHUA · 2013 to 2025
$1.8M
Intramural NIH HHS Z99 EY999999
6 · The paper itself

Abstract

Recombinant adeno-associated virus (rAAV) has been widely used for gene delivery in animal models and successfully applied in clinical trials for treating inherited retinal disease. Although subretinal delivery of AAVs can effectively transduce photoreceptors and/or retinal pigmental epithelium (RPE), cells most affected by inherited retinal diseases, the procedure is invasive and complicated, and only delivers the gene to a limited retinal area. AAVs can also be delivered intravitreally to the retina, a much less invasive nonsurgical procedure. However, intravitreal administration of non-modified AAV serotypes tends to transduce only ganglion cells and inner nuclear layer cells. To date, most non-modified AAV serotypes that have been identified are incapable of efficiently transducing photoreceptors and/or RPE when delivered intravitreally. In this study, we investigate the retinal tropism of AAVrh10 vector administered by intravitreal injection to mouse, rat, and rabbit eyes. Our results demonstrate that AAVrh10 is capable of transducing not only inner retinal cells, but also outer retinal cells in all three species, though the transduction efficiency in rabbit was low. In addition, AAVrh10 preferentially transduced outer retinal cells in mouse models of retinal disease. Therefore, AAVrh10 vector could be a useful candidate to intravitreally deliver genes to photoreceptor and RPE cells.

Indexed as

RetinaAnimalsCytomegalovirusDependovirusGenetic VectorsGreen Fluorescent ProteinsIntravitreal InjectionsMaleMicePhotoreceptor CellsRabbitsRatsRats, Sprague-DawleyRetinal DiseasesTransduction, GeneticViral TropismGreen Fluorescent Proteins

Identifiers

PMID31308478
PMCPMC11388630
OpenAlexW2962397111

What OpenQuestion holds

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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.