Trial reportInvestigative ophthalmology & visual science2014
Pseudo-fovea formation after gene therapy for RPE65-LCA.
Trial report in Investigative ophthalmology & visual science, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT00481546 (Phase I Trial of Ocular Subretinal Injection of a Recombinant Adeno-Associated Virus), which is not on this map. Cited by 26 papers, 1 of them a synthesis that pooled 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.
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.
Phase I Trial of Ocular Subretinal Injection of a Recombinant Adeno-Associated Virus (rAAV2-CBSB-hRPE65) Gene Vector to Patients With Retinal Disease Due to RPE65 Mutations (Clinical Trials of Gene Therapy for Leber Congenital Amaurosis)
Who cites it
26 citing papers in PubMed, 1 synthesis or guideline pooled it, 38 citations in OpenAlex.
- Safety and Efficacy of Adeno-Associated Viral Gene Therapy in Patients With Retinal Degeneration: A Systematic Review and Meta-Analysis.Translational vision science & technology · 2023Pooled it
- Restoration of Cone Sensitivity to Individuals with Congenital Photoreceptor Blindness within the Phase 1/2 Sepofarsen Trial.Ophthalmology science · 2022Article
- Cortical Visual Mapping following Ocular Gene Augmentation Therapy for Achromatopsia.The Journal of neuroscience : the official journal of the Society for Neuroscience · 2021Article
- Durable vision improvement after a single treatment with antisense oligonucleotide sepofarsen: a case report.Nature medicine · 2021Article
- Clinical Perspective: Treating RPE65-Associated Retinal Dystrophy.Molecular therapy : the journal of the American Society of Gene Therapy · 2021Review
- Mapping Visual Field Defects With fMRI - Impact of Approach and Experimental Conditions.Frontiers in neuroscience · 2021Article
- A Glossary for ''Pseudo'' Conditions in Ophthalmology.Beyoglu eye journal · 2020Review
- Leber Congenital Amaurosis (LCA): Potential for Improvement of Vision.Investigative ophthalmology & visual science · 2019Article
- Outcome Measures Used in Ocular Gene Therapy Trials: A Scoping Review of Current Practice.Frontiers in pharmacology · 2019Article
- Clinical applications of retinal gene therapies.Precision clinical medicine · 2018Review
- Pupillary Light Reflexes in Severe Photoreceptor Blindness Isolate the Melanopic Component of Intrinsically Photosensitive Retinal Ganglion Cells.Investigative ophthalmology & visual science · 2017Article
- Postretinal Structure and Function in Severe Congenital Photoreceptor Blindness Caused by Mutations in the GUCY2D Gene.Investigative ophthalmology & visual science · 2017Article
- Timing of Antioxidant Gene Therapy: Implications for Treating Dry AMD.Investigative ophthalmology & visual science · 2017Article
- Review
- Outer Plexiform Layer Structures Are Not Altered Following AAV-Mediated Gene Transfer in Healthy Rat Retina.Frontiers in neurology · 2017Article
- Visual Cortex Plasticity Following Peripheral Damage To The Visual System: fMRI Evidence.Current neurology and neuroscience reports · 2016Review
- Developing an Outcome Measure With High Luminance for Optogenetics Treatment of Severe Retinal Degenerations and for Gene Therapy of Cone Diseases.Investigative ophthalmology & visual science · 2016Article
- Let There Be Light: Gene and Cell Therapy for Blindness.Human gene therapy · 2016Review
- Non-viral therapeutic approaches to ocular diseases: An overview and future directions.Journal of controlled release : official journal of the Controlled Release Society · 2015Review
- Gene Augmentation Therapy Restores Retinal Function and Visual Behavior in a Sheep Model of CNGA3 Achromatopsia.Molecular therapy : the journal of the American Society of Gene Therapy · 2015Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
9 authors at 3 institutions in 1 country.
Funding
Abstract
purposeThe purpose of this study was to evaluate fixation location and oculomotor characteristics of 15 patients with Leber congenital amaurosis (LCA) caused by RPE65 mutations (RPE65-LCA) who underwent retinal gene therapy.
methodsEye movements were quantified under infrared imaging of the retina while the subject fixated on a stationary target. In a subset of patients, letter recognition under retinal imaging was performed. Cortical responses to visual stimulation were measured using functional magnetic resonance imaging (fMRI) in two patients before and after therapy.
resultsAll patients were able to fixate on a 1° diameter visible target in the dark. The preferred retinal locus of fixation was either at the anatomical fovea or at an extrafoveal locus. There were a wide range of oculomotor abnormalities. Natural history showed little change in oculomotor abnormalities if target illuminance was increased to maintain target visibility as the disease progressed. Eleven of 15 study eyes treated with gene therapy showed no differences from baseline fixation locations or instability over an average of follow-up of 3.5 years. Four of 15 eyes developed new pseudo-foveas in the treated retinal regions 9 to 12 months after therapy that persisted for up to 6 years; patients used their pseudo-foveas for letter identification. fMRI studies demonstrated that preservation of light sensitivity was restricted to the cortical projection zone of the pseudo-foveas.
conclusionsThe slow emergence of pseudo-foveas many months after the initial increases in light sensitivity points to a substantial plasticity of the adult visual system and a complex interaction between it and the progression of underlying retinal disease. The visual significance of pseudo-foveas suggests careful consideration of treatment zones for future gene therapy trials. (ClinicalTrials.gov number, NCT00481546.).
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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.