Evidence map›Paper›PMID 42411064›Full record

ArticleHuman gene therapy2026

Evaluation of the Toxicity and Efficacy of an Adeno-Associated Viral Vector Expressing

Alexa Gray, Jennifer C Kwok, Yu Sato, Charles-Antoine Assenmacher, Mayur Choudhary, Ash Jayagopal, Peiying Hua, Gui-Shuang Ying, Malgorzata Swider, Alexander Sumaroka and 3 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Human gene therapy, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

13 authors.

Alexa GrayDivision of Experimental Retinal Therapies, Department of Clinical Sciences & Advanced Medicine, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
Jennifer C KwokDivision of Experimental Retinal Therapies, Department of Clinical Sciences & Advanced Medicine, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
Yu SatoDivision of Experimental Retinal Therapies, Department of Clinical Sciences & Advanced Medicine, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.ORCID 0000-0002-5940-1349
Charles-Antoine AssenmacherComparative Pathology Core, Department of Pathobiology, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
Mayur ChoudharyOpus Genetics, Inc., Durham, North Carolina, USA.
Ash JayagopalOpus Genetics, Inc., Durham, North Carolina, USA.
Peiying HuaCenter for Hereditary Retinal Degenerations, Department of Ophthalmology, Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
Gui-Shuang YingCenter for Hereditary Retinal Degenerations, Department of Ophthalmology, Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
Malgorzata SwiderCenter for Hereditary Retinal Degenerations, Department of Ophthalmology, Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
Alexander SumarokaCenter for Hereditary Retinal Degenerations, Department of Ophthalmology, Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
Artur V CideciyanCenter for Hereditary Retinal Degenerations, Department of Ophthalmology, Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
Aguirre D GustavoDivision of Experimental Retinal Therapies, Department of Clinical Sciences & Advanced Medicine, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
William A BeltranDivision of Experimental Retinal Therapies, Department of Clinical Sciences & Advanced Medicine, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.ORCID 0000-0002-1127-4774

Funding

MODELS OF HEREDITY RETINAL DEGENERATIONR01EY006855 · NEI · UNIVERSITY OF PENNSYLVANIA · PI GUSTAVO David AGUIRRE, William A. Beltran · 1992 to 2026
$20.7M
Scientific TransparencyP30EY001583 · NEI · UNIVERSITY OF PENNSYLVANIA · PI CLAIRE H MITCHELL · 1985 to 2026
$19.5M
NEI NIH HHS P30 EY001583NEI NIH HHS R01 EY006855
6 · The paper itself

Abstract

To treat patients affected with bestrophinopathies caused by mutations in the

Indexed as

BestrophinsDependovirusEye Diseases, HereditaryGenetic TherapyGenetic VectorsRetinal DiseasesAnimalsDisease Models, AnimalDogsElectroretinographyGene Therapy AgentsHumansMutationRetinaBEST1 protein, humanBestrophinsautosomal recessive bestrophinopathy (ARB)best vitelliform macular dystrophy (BVMD)gene therapyIND-enabling studyphotoreceptor function

Identifiers

PMID42411064
PMCPMC13401768

What Socratic holds

Textmetadata
LicenceTDM
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 Socratic graph.