ArticleGenetic vaccines and therapy2004
Recombinant adeno-associated virus type 2-mediated gene delivery into the
Article in Genetic vaccines and therapy, 2004. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 48 papers.
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
48 citing papers in PubMed.
- Results at 5 Years After Gene Therapy for RPE65-Deficient Retinal Dystrophy.Human gene therapy · 2018Trial
- Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics.Proceedings of the National Academy of Sciences of the United States of America · 2008Trial
- Gene and Stem Cell-Based Therapies for Retinal Degenerative Diseases: Update, Challenges, and Future Directions.Stem cell reviews and reports · 2025Review
- One down but many more to go: the state of gene therapy for inherited retinal disease.Regenerative medicine · 2025Review
- A non-surgical method for subretinal delivery by trans-scleral microneedle injection.Bioengineering & translational medicine · 2025Article
- Partial rescue of the full-field electroretinogram in patients with RPE65-related retinal dystrophy following gene augmentation therapy with voretigene neparvovec-rzyl.Documenta ophthalmologica. Advances in ophthalmology · 2024Article
- Gene therapy in retinal diseases: A review.Indian journal of ophthalmology · 2021Review
- Article
- Voretigene Neparvovec in Retinal Diseases: A Review of the Current Clinical Evidence.Clinical ophthalmology (Auckland, N.Z.) · 2020Review
- Gene Therapy in Retinal Dystrophies.International journal of molecular sciences · 2019Review
- Molecular Engineering of Adeno-Associated Virus Capsid Improves Its Therapeutic Gene Transfer in Murine Models of Hemophilia and Retinal Degeneration.Molecular pharmaceutics · 2019Article
- Therapeutic Benefits from Nanoparticles: The Potential Significance of Nanoscience in Retinal Degenerative Diseases.Journal of molecular biology & therapeutics · 2019Article
- Targeted Multifunctional Lipid ECO Plasmid DNA Nanoparticles as Efficient Non-viral Gene Therapy for Leber's Congenital Amaurosis.Molecular therapy. Nucleic acids · 2017Article
- Outer Plexiform Layer Structures Are Not Altered Following AAV-Mediated Gene Transfer in Healthy Rat Retina.Frontiers in neurology · 2017Article
- Cell-Specific Promoters Enable Lipid-Based Nanoparticles to Deliver Genes to Specific Cells of the Retina In Vivo.Theranostics · 2016Article
- Lipid Nanoparticles for Ocular Gene Delivery.Journal of functional biomaterials · 2015Review
- Dog models for blinding inherited retinal dystrophies.Human gene therapy. Clinical development · 2015Review
- The Status of RPE65 Gene Therapy Trials: Safety and Efficacy.Cold Spring Harbor perspectives in medicine · 2015Review
- Trans-Corneal Subretinal Injection in Mice and Its Effect on the Function and Morphology of the Retina.PloS one · 2015Article
- Identification of key residues determining isomerohydrolase activity of human RPE65.The Journal of biological chemistry · 2014Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
8 authors.
Funding
Abstract
backgroundLeber's congenital amaurosis (LCA) is a severe form of retinal dystrophy. Mutations in the RPE65 gene, which is abundantly expressed in retinal pigment epithelial (RPE) cells, account for approximately 10-15% of LCA cases. In this study we used the high turnover, and rapid breeding and maturation time of the
methodsrAAV.RPE65 was injected into the subretinal space of
resultsrAAV-mediated RPE65 expression was detected for up to 18 months post injection. The delivery of rAAV.RPE65 to
conclusionThis work demonstrated the potential benefits of using the
Identifiers
What OpenQuestion holds
Registered trials
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.