ArticleHuman gene therapy2020
Toxicity and Efficacy Evaluation of an Adeno-Associated Virus Vector Expressing Codon-Optimized
Erratum issuedOpen access · greenAbstract read
In one paragraph
Article in Human gene therapy, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 28 papers, 2 of them syntheses that pooled it.
0numbers the graph read from it
0cells of the map it votes in
28citing papers in PubMed, 2 pooled it
1.7field-weighted citation impact, top 16% of its field
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.
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.
Who cites it
28 citing papers in PubMed, 2 syntheses or guidelines pooled it, 36 citations in OpenAlex.
- Efficacy and safety of AAV RPGR gene therapy in X-linked retinitis pigmentosa: a systematic review and meta-analysis.Journal of translational medicine · 2026Pooled it
- Retinal Viral Gene Therapy: Impact of Route of Administration on Serious Adverse Events-A Systematic Review.Clinical & experimental ophthalmology · 2025Pooled it
- Evaluation of the Toxicity and Efficacy of an Adeno-Associated Viral Vector ExpressingHuman gene therapy · 2026Article
- Single-cell and spatial transcriptomic analyses of gene therapy-associated retinal inflammation in non-human primates.Molecular therapy. Advances · 2026Article
- A gel-forming antioxidant eye drop for photoreceptor protection in retinitis pigmentosa.Drug delivery and translational research · 2026Article
- Advances in RPGR gene therapy for X‑linked retinitis pigmentosa: From preclinical insights to clinical application (Review).International journal of molecular medicine · 2026Review
- A Comprehensive Review of Clinically Applied Adeno-Associated Virus-Based Gene Therapies for Ocular Disease.Human gene therapy · 2025Review
- Canine models of inherited retinal diseases: from neglect to well-recognized translational value.Mammalian genome : official journal of the International Mammalian Genome Society · 2025Review
- Review
- Pre-Clinical and Clinical Advances in Gene Therapy of X-Linked Retinitis Pigmentosa: Hope on the Horizon.Journal of clinical medicine · 2025Review
- Immune responses in retinal gene therapy: challenges, mechanisms, and future strategies.Frontiers in immunology · 2025Review
- Recent progress of principal techniques used in the study of Müller glia reprogramming in mice.Cell regeneration (London, England) · 2024Review
- An amplification-free CRISPR-Cas12a assay for titer determination and composition analysis of the rAAV genome.Molecular therapy. Methods & clinical development · 2024Article
- Lipid nanoparticle technology-mediated therapeutic gene manipulation in the eyes.Molecular therapy. Nucleic acids · 2024Review
- Synthetic biology approach revealed enhancement in haeme oxygenase-1 gene expression by codon pair optimization while reduction by codon deoptimization.Annals of medicine and surgery (2012) · 2024Article
- Clinical sequencing of the retinitis pigmentosa geneMolecular vision · 2024Article
- Review
- Cone Structure and Function in RPGR- and USH2A-Associated Retinal Degeneration.American journal of ophthalmology · 2023Article
- Gene Therapy for Retinal Degenerative Diseases: Progress, Challenges, and Future Directions.Investigative ophthalmology & visual science · 2023Review
- Adeno-Associated Virus (AAV) - Based Gene Therapies for Retinal Diseases: Where are We?The application of clinical genetics · 2023Review
Corrections and comments
- Erratum issuedCorrection to:2020
Authors and funding
18 authors at 3 institutions in 1 country.
Valérie L DufourDivision of Experimental Retinal Therapies, Department of Clinical Sciences & Advanced Medicine, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.
Artur V CideciyanDepartment of Ophthalmology, Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.
Guo-Jie YeApplied Genetic Technologies Corporation, Alachua, Florida.
Chunjuan SongApplied Genetic Technologies Corporation, Alachua, Florida.
Adrian TimmersApplied Genetic Technologies Corporation, Alachua, Florida.
Perry L HabeckerDepartment of Pathobiology, New Bolton Center, School of Veterinary Medicine, University of Pennsylvania, Kennett Square, Pennsylvania.
Wei PanDepartment of Ophthalmology, Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.
Nicole M WeinsteinDepartment of Pathobiology, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.
Malgorzata SwiderDepartment of Ophthalmology, Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.
Amy C DurhamDepartment of Pathobiology, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.
Gui-Shuang YingDepartment of Ophthalmology, Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.
Paulette M RobinsonApplied Genetic Technologies Corporation, Alachua, Florida.
Samuel G JacobsonDepartment of Ophthalmology, Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.
David R KnopApplied Genetic Technologies Corporation, Alachua, Florida.
Jeffrey D ChulayApplied Genetic Technologies Corporation, Alachua, Florida.
Mark S ShearmanApplied Genetic Technologies Corporation, Alachua, Florida.
Gustavo D AguirreDivision of Experimental Retinal Therapies, Department of Clinical Sciences & Advanced Medicine, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.
William A BeltranDivision of Experimental Retinal Therapies, Department of Clinical Sciences & Advanced Medicine, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.
Applied Genetic Technologies (United States) · USUniversity of Pennsylvania · USPenn Presbyterian Medical Center · US
Funding
MODELS OF HEREDITY RETINAL DEGENERATIONR01EY006855 · NEI · UNIVERSITY OF PENNSYLVANIA · PI GUSTAVO David AGUIRRE, William A. Beltran · 1992 to 2026
$20.7MScientific TransparencyP30EY001583 · NEI · UNIVERSITY OF PENNSYLVANIA · PI CLAIRE H MITCHELL · 1985 to 2026
$19.5MTranslational Research for Retinal Degeneration TherapiesR01EY017549 · NEI · UNIVERSITY OF PENNSYLVANIA · PI BELTRAN, WILLIAM A. · 2007 to 2024
$14.1MNEI NIH HHS P30 EY001583NEI NIH HHS R01 EY006855NEI NIH HHS R01 EY017549
Abstract
Applied Genetic Technologies Corporation (AGTC) is developing a recombinant adeno-associated virus (rAAV) vector AGTC-501, also designated rAAV2tYF-GRK1-
Indexed as
Genes, X-LinkedGenetic TherapyAnimalsBiomarkersBiopsyCell LineCodonDependovirusDogsElectroretinographyEye ProteinsGenetic VectorsHumansImmunohistochemistryMutationRetinitis PigmentosaBiomarkersCodonEye ProteinsRPGR protein, humangene therapyretinal degenerationRPGRXLRP
Identifiers
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.