Evidence map›Paper›PMID 25092770›Full record

ArticleEMBO molecular medicine2014

Efficient transduction and optogenetic stimulation of retinal bipolar cells by a synthetic adeno-associated virus capsid and promoter.

Therese Cronin, Luk H Vandenberghe, Péter Hantz, Josephine Juttner, Andreas Reimann, Agota-Enikő Kacsó, Rachel M Huckfeldt, Volker Busskamp, Hubertus Kohler, Pamela S Lagali and 2 more

Open access · goldAbstract read
In one paragraph

Article in EMBO molecular medicine, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 108 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
108citing papers in PubMed, 1 pooled it
7.8field-weighted citation impact, top 2% 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

108 citing papers in PubMed, 1 synthesis or guideline pooled it, 182 citations in OpenAlex.

  1. Pooled it
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  6. Optogenetic vision restoration in the face of secondary and tertiary remodeling in the rd1 mouse retina.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Article
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  16. Article
  17. Optogenetic Vision Restoration.Cold Spring Harbor perspectives in medicine · 2024
    Review
  18. Article
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  20. Article

48 more citing papers are in PubMed but not listed here.

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

12 authors at 7 institutions in 4 countries.

Therese CroninCenter for Advanced Retinal and Ophthalmic Therapeutics, F.M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA, USA Friedrich Miescher Institute for Biomedical Research, Basel, Switzerland jebennet@mail.med.upenn.edu therese.cronin@fmi.ch.
Luk H VandenbergheCenter for Advanced Retinal and Ophthalmic Therapeutics, F.M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA, USA Schepens Eye Research Institute, Massachusetts Eye and Ear Infirmary, Harvard Medical School, Boston, MA, USA.
Péter HantzFriedrich Miescher Institute for Biomedical Research, Basel, Switzerland.
Josephine JuttnerFriedrich Miescher Institute for Biomedical Research, Basel, Switzerland.
Andreas ReimannFriedrich Miescher Institute for Biomedical Research, Basel, Switzerland.
Agota-Enikő KacsóFaculty of Physics, Babes-Bolyai University, Cluj-Napoca, Romania.
Rachel M HuckfeldtCenter for Advanced Retinal and Ophthalmic Therapeutics, F.M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA, USA.
Volker BusskampFriedrich Miescher Institute for Biomedical Research, Basel, Switzerland Genetics Department, Harvard Medical School, Boston, MA, USA.
Hubertus KohlerFriedrich Miescher Institute for Biomedical Research, Basel, Switzerland.
Pamela S LagaliFriedrich Miescher Institute for Biomedical Research, Basel, Switzerland Regenerative Medicine Program, Ottawa Hospital Research Institute, Ottawa, ON, Canada.
Botond RoskaFriedrich Miescher Institute for Biomedical Research, Basel, Switzerland.
Jean BennettCenter for Advanced Retinal and Ophthalmic Therapeutics, F.M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA, USA jebennet@mail.med.upenn.edu therese.cronin@fmi.ch.
Friedrich Miescher Institute · CHUniversity of Pennsylvania · USBabeș-Bolyai University · ROHarvard University · USMassachusetts Eye and Ear Infirmary · USOttawa Hospital · CAPenn Presbyterian Medical Center · US

Funding

Scientific TransparencyP30EY001583 · NEI · UNIVERSITY OF PENNSYLVANIA · PI William A. Beltran · 1985 to 2026
$19.5M
Therapeutic approaches for ABCA4-associated disordersR24EY019861 · NEI · COLUMBIA UNIVERSITY HEALTH SCIENCES · PI ALLIKMETS, RANDO L · 2011 to 2015
$7.2M
Broad Spectrum Molecular Therapy for Blinding Retina DisordersDP1EY023177 · NEI · UNIVERSITY OF PENNSYLVANIA · PI BENNETT, JEAN · 2012 to 2015
$3.2M
Broad Spectrum Molecular Therapy for Blinding Retina DisordersDP1OD008267 · OD · UNIVERSITY OF PENNSYLVANIA · PI BENNETT, JEAN · 2011 to 2011
$800k
NCCDPHP CDC HHS 8DP1 EY023177NEI NIH HHS 1R24EY019861‐01A1NEI NIH HHS DP1 EY023177NEI NIH HHS P30 EY001583NEI NIH HHS R24 EY019861NIH HHS DP1 OD008267
6 · The paper itself

Abstract

In this report, we describe the development of a modified adeno-associated virus (AAV) capsid and promoter for transduction of retinal ON-bipolar cells. The bipolar cells, which are post-synaptic to the photoreceptors, are important retinal targets for both basic and preclinical research. In particular, a therapeutic strategy under investigation for advanced forms of blindness involves using optogenetic molecules to render ON-bipolar cells light-sensitive. Currently, delivery of adequate levels of gene expression is a limiting step for this approach. The synthetic AAV capsid and promoter described here achieves high level of optogenetic transgene expression in ON-bipolar cells. This evokes high-frequency (~100 Hz) spiking responses in ganglion cells of previously blind, rd1, mice. Our vector is a promising vehicle for further development toward potential clinical use.

Indexed as

AnimalsDependovirusGenetic VectorsHEK293 CellsHumansMiceMice, Inbred C57BLMutagenesis, Site-DirectedPromoter Regions, GeneticRetinal Bipolar CellsTransduction, Geneticadeno‐associated viruscapsid librarymulti‐electrode arrayoptogeneticspromoter optimization

Identifiers

PMID25092770
PMCPMC4197864
OpenAlexW2140320479

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.