ReviewGene therapy2012
Gene supplementation therapy for recessive forms of inherited retinal dystrophies.
Review in Gene therapy, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 27 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
27 citing papers in PubMed, 60 citations in OpenAlex.
- New Perspectives in Stem Cell Transplantation and Associated Therapies to Treat Retinal Diseases: From Gene Editing to 3D Bioprinting.Stem cell reviews and reports · 2024Review
- Role of Notch1 Signaling Pathway in Small Cell Lung Carcinoma.Iranian journal of pathology · 2024Review
- Article
- Viral Vector-Based Gene Therapy.International journal of molecular sciences · 2023Review
- Leber congenital amaurosis/early-onset severe retinal dystrophy: current management and clinical trials.The British journal of ophthalmology · 2022Review
- Functional Availability of ON-Bipolar Cells in the Degenerated Retina: Timing and Longevity of an Optogenetic Gene Therapy.International journal of molecular sciences · 2021Article
- Gene Editing Preserves Visual Functions in a Mouse Model of Retinal Degeneration.Frontiers in neuroscience · 2019Article
- Targeted elimination of mutant mitochondrial DNA in MELAS-iPSCs by mitoTALENs.Protein & cell · 2018Article
- Infantile Neuroaxonal Dystrophy: Diagnosis and Possible Treatments.Frontiers in genetics · 2018Article
- Progress in Gene Therapy to Prevent Retinal Ganglion Cell Loss in Glaucoma and Leber's Hereditary Optic Neuropathy.Neural plasticity · 2018Review
- Inherited Retinal Disease Therapies Targeting Precursor Messenger Ribonucleic Acid.Vision (Basel, Switzerland) · 2017Review
- A fully organic retinal prosthesis restores vision in a rat model of degenerative blindness.Nature materials · 2017Article
- Harnessing the Potential of Human Pluripotent Stem Cells and Gene Editing for the Treatment of Retinal Degeneration.Current stem cell reports · 2017Review
- Genetic manipulation for inherited neurodegenerative diseases: myth or reality?The British journal of ophthalmology · 2016Review
- Treatment of retinitis pigmentosa due to MERTK mutations by ocular subretinal injection of adeno-associated virus gene vector: results of a phase I trial.Human genetics · 2016Article
- Inhibition of pathological brain angiogenesis through systemic delivery of AAV vector expressing soluble FLT1.Gene therapy · 2015Article
- Virally mediated Kcnq1 gene replacement therapy in the immature scala media restores hearing in a mouse model of human Jervell and Lange-Nielsen deafness syndrome.EMBO molecular medicine · 2015Article
- Promising and delivering gene therapies for vision loss.Vision research · 2015Review
- Gene expression changes during retinal development and rod specification.Molecular vision · 2015Article
- Retinal prostheses: progress toward the next generation implants.Frontiers in neuroscience · 2015Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors at 1 institution in 1 country.
Funding
Abstract
Over the last decade, gene supplementation therapy for inherited retinal degeneration has come of age. Early proof-of-concept studies in animal models of disease showed modest, but genuine improvements in retinal function and/or survival. Further development of the vectors used for gene transfer to the retina has led to better treatment efficacy in a wide variety of animal models, leading in 2008 to the initiation of three clinical trials for Leber congenital amaurosis caused by retinal pigment epithelium 65 deficiency. The results from these trials suggest that the treatment of inherited retinal dystrophy by gene therapy can be safe and effective. Here, we examine the progress of gene supplementation therapy in the retina, and discuss the potential for using gene therapy to treat different forms of inherited retinal degeneration.
Indexed as
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.