ReviewGene therapy2012
Gene-based therapies for dominantly inherited retinopathies.
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 24 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
24 citing papers in PubMed.
- Novel compound heterozygousExperimental and therapeutic medicine · 2025Article
- Non-Viral Delivery Systems to Transport Nucleic Acids for Inherited Retinal Disorders.Pharmaceuticals (Basel, Switzerland) · 2025Review
- Biomarkers and Novel Therapies of Diabetic Neuropathy: An Updated Review.Current pharmaceutical biotechnology · 2025Review
- Update on Clinical Trial Endpoints in Gene Therapy Trials for Inherited Retinal Diseases.Journal of clinical medicine · 2024Review
- PRPF31-retinitis pigmentosa: Challenges and opportunities for clinical translation.Vision research · 2023Review
- Gene augmentation for autosomal dominant retinitis pigmentosa using rhodopsin genomic loci nanoparticles in the P23HGene therapy · 2023Article
- CRISPR genome surgery in a novel humanized model for autosomal dominant retinitis pigmentosa.Molecular therapy : the journal of the American Society of Gene Therapy · 2022Article
- Advancing precision medicines for ocular disorders: Diagnostic genomics to tailored therapies.Frontiers in medicine · 2022Review
- Gene therapy for inherited retinal diseases.Annals of translational medicine · 2021Review
- Curative gene therapies for rare diseases.Journal of community genetics · 2021Article
- Gene-Based Therapeutics for Inherited Retinal Diseases.Frontiers in genetics · 2021Review
- Molecular Therapies for Inherited Retinal Diseases-Current Standing, Opportunities and Challenges.Genes · 2019Review
- Genome Editing as a Treatment for the Most Prevalent Causative Genes of Autosomal Dominant Retinitis Pigmentosa.International journal of molecular sciences · 2019Review
- Phenotypic characterization of P23H and S334ter rhodopsin transgenic rat models of inherited retinal degeneration.Experimental eye research · 2018Article
- Guiding Lights in Genome Editing for Inherited Retinal Disorders: Implications for Gene and Cell Therapy.Neural plasticity · 2018Review
- Gene therapy approaches for the treatment of retinal disorders.Discovery medicine · 2016Review
- Genetic manipulation for inherited neurodegenerative diseases: myth or reality?The British journal of ophthalmology · 2016Review
- Defining the catalytic activity of nanoceria in the P23H-1 rat, a photoreceptor degeneration model.PloS one · 2015Article
- Adaptive optics imaging of the retina.Indian journal of ophthalmology · 2014Review
- GPCR: G protein complexes--the fundamental signaling assembly.Amino acids · 2013Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
In light of the elucidation of the molecular pathogenesis of some dominantly inherited retinal degenerations over the past two decades, it is timely to explore possible means of therapeutic intervention for such diseases. However, the presence of significant levels of intergenic and intragenic genetic heterogeneity in this group of dominant conditions represents a barrier to the development of therapies focused on correcting the primary genetic defect. More than 60 genes have been implicated in dominant retinopathies and indeed over 150 different mutations in the rhodopsin gene alone have been identified in patients with autosomal dominant retinitis pigmentosa. Employing next-generation sequencing to characterise populations of retinal degeneration patients genetically over the coming years will beyond doubt serve to highlight further the immense genetic heterogeneity inherent in this group of disorders. Such diversity in genetic aetiologies has promoted the search for therapeutic solutions for dominantly inherited retinopathies that are independent of disease-causing mutations. The various approaches being considered to provide mutation-independent therapies for these dominant conditions will be discussed in the review, as will the preclinical data supporting the further development of such strategies.
Indexed as
Identifiers
22089493What 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.