Evidence map›Paper›PMID 35604672›Full record

ArticleTranslational vision science & technology2022

Restoration of Vision and Retinal Responses After Adeno-Associated Virus-Mediated Optogenetic Therapy in Blind Dogs.

Sergei Nikonov, Puya Aravand, Arkady Lyubarsky, Roman Nikonov, Angela J Luo, Zhangyong Wei, Albert M Maguire, Nicholas T Phelps, Ivan Shpylchak, Keirnan Willett and 4 more

Open access · goldAbstract read
In one paragraph

Article in Translational vision science & technology, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
15citing papers in PubMed, 1 pooled it
1.6field-weighted citation impact, top 18% 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

15 citing papers in PubMed, 1 synthesis or guideline pooled it, 18 citations in OpenAlex.

  1. Pooled it
  2. Review
  3. Article
  4. 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
  5. Article
  6. Review
  7. Article
  8. Review
  9. Optogenetic Vision Restoration.Cold Spring Harbor perspectives in medicine · 2024
    Review
  10. Review
  11. Article
  12. Article
  13. Review
  14. Review
  15. Optogenetic Therapy for Visual Restoration.International journal of molecular sciences · 2022
    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

14 authors at 2 institutions in 1 country.

Sergei NikonovCenter for Advanced Retinal and Ocular Therapeutics (CAROT) and F.M. Kirby Center for Molecular Ophthalmology Scheie Eye Institute, University of Pennsylvania, Perelman School of Medicine, Philadelphia, PA, USA.
Puya AravandCenter for Advanced Retinal and Ocular Therapeutics (CAROT) and F.M. Kirby Center for Molecular Ophthalmology Scheie Eye Institute, University of Pennsylvania, Perelman School of Medicine, Philadelphia, PA, USA.
Arkady LyubarskyCenter for Advanced Retinal and Ocular Therapeutics (CAROT) and F.M. Kirby Center for Molecular Ophthalmology Scheie Eye Institute, University of Pennsylvania, Perelman School of Medicine, Philadelphia, PA, USA.
Roman NikonovCenter for Advanced Retinal and Ocular Therapeutics (CAROT) and F.M. Kirby Center for Molecular Ophthalmology Scheie Eye Institute, University of Pennsylvania, Perelman School of Medicine, Philadelphia, PA, USA.
Angela J LuoCenter for Advanced Retinal and Ocular Therapeutics (CAROT) and F.M. Kirby Center for Molecular Ophthalmology Scheie Eye Institute, University of Pennsylvania, Perelman School of Medicine, Philadelphia, PA, USA.
Zhangyong WeiCenter for Advanced Retinal and Ocular Therapeutics (CAROT) and F.M. Kirby Center for Molecular Ophthalmology Scheie Eye Institute, University of Pennsylvania, Perelman School of Medicine, Philadelphia, PA, USA.
Albert M MaguireCenter for Advanced Retinal and Ocular Therapeutics (CAROT) and F.M. Kirby Center for Molecular Ophthalmology Scheie Eye Institute, University of Pennsylvania, Perelman School of Medicine, Philadelphia, PA, USA.
Nicholas T PhelpsCenter for Advanced Retinal and Ocular Therapeutics (CAROT) and F.M. Kirby Center for Molecular Ophthalmology Scheie Eye Institute, University of Pennsylvania, Perelman School of Medicine, Philadelphia, PA, USA.
Ivan ShpylchakCenter for Advanced Retinal and Ocular Therapeutics (CAROT) and F.M. Kirby Center for Molecular Ophthalmology Scheie Eye Institute, University of Pennsylvania, Perelman School of Medicine, Philadelphia, PA, USA.
Keirnan WillettCenter for Advanced Retinal and Ocular Therapeutics (CAROT) and F.M. Kirby Center for Molecular Ophthalmology Scheie Eye Institute, University of Pennsylvania, Perelman School of Medicine, Philadelphia, PA, USA.
Tomas S AlemanCenter for Advanced Retinal and Ocular Therapeutics (CAROT) and F.M. Kirby Center for Molecular Ophthalmology Scheie Eye Institute, University of Pennsylvania, Perelman School of Medicine, Philadelphia, PA, USA.
Rachel M HuckfeldtCenter for Advanced Retinal and Ocular Therapeutics (CAROT) and F.M. Kirby Center for Molecular Ophthalmology Scheie Eye Institute, University of Pennsylvania, Perelman School of Medicine, Philadelphia, PA, USA.
Pavitra S RamachandranCenter for Advanced Retinal and Ocular Therapeutics (CAROT) and F.M. Kirby Center for Molecular Ophthalmology Scheie Eye Institute, University of Pennsylvania, Perelman School of Medicine, Philadelphia, PA, USA.
Jean BennettCenter for Advanced Retinal and Ocular Therapeutics (CAROT) and F.M. Kirby Center for Molecular Ophthalmology Scheie Eye Institute, University of Pennsylvania, Perelman School of Medicine, Philadelphia, PA, USA.
Penn Presbyterian Medical Center · USUniversity of Pennsylvania · US

Funding

Scientific TransparencyP30EY001583 · NEI · UNIVERSITY OF PENNSYLVANIA · PI CLAIRE H MITCHELL · 1985 to 2026
$19.5M
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
NEI NIH HHS DP1 EY023177NEI NIH HHS P30 EY001583NIH HHS DP1 OD008267
6 · The paper itself

Abstract

Purpose: Optogenetic gene therapy to render remaining retinal cells light-sensitive in end-stage retinal degeneration is a promising strategy for treatment of individuals blind because of a variety of different inherited retinal degenerations. The clinical trials currently in progress focus on delivery of optogenetic genes to ganglion cells. Delivery of optogenetic molecules to cells in the outer neural retina is predicted to be even more advantageous because it harnesses more of the retinal circuitry. However, this approach has not yet been tested in large animal models. For this reason, we evaluated the safety and efficacy of optogenetic therapy targeting remaining diseased cone photoreceptors in the Rcd1 dog model of retinitis pigmentosa. Methods: Imaging and measures of retinal function and functional vision were carried out, as well as terminal studies evaluating multi-electrode array recordings and histology. Results: Animals remained healthy and active throughout the study and showed improved retinal and visual function as assessed by electroretinography and visual-evoked potentials, improved navigational vision, and improved function of cone photoreceptors and the downstream retinal circuitry. Conclusions: The findings demonstrate that an optogenetic approach targeting the outer retina in a blind large animal model can partially restore vision. Translational Relevance: This work has translational relevance because the approach could potentially be extrapolated to treat humans who are totally blind because of retinal degenerative disease.

Indexed as

DependovirusRetinal DegenerationAnimalsDogsOptogeneticsRetinaRetinal Cone Photoreceptor CellsVision, Ocular

Identifiers

PMID35604672
PMCPMC9145127
OpenAlexW4281398263

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.