ReviewCold Spring Harbor perspectives in medicine2014
Gene therapy for choroideremia using an adeno-associated viral (AAV) vector.
Review in Cold Spring Harbor perspectives in medicine, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 26 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
26 citing papers in PubMed, 58 citations in OpenAlex.
- Gene therapy in patients with choroideremia: a meta-analysis of clinical trials.Graefe's archive for clinical and experimental ophthalmology = Albrecht von Graefes Archiv fur klinische und experimentelle Ophthalmologie · 2026Article
- Anticodon-edited transfer RNAs (ACE-tRNAs) encoded as therapeutic nonviral minimal DNA vectors.Nucleic acids research · 2026Article
- Anticodon Edited Transfer RNAs (ACE-tRNAs) Encoded as Therapeutic Nonviral Minimal DNA Vectors.bioRxiv : the preprint server for biology · 2025Article
- Revolutionizing Veterinary Health with Viral Vector-Based Vaccines.Indian journal of microbiology · 2024Review
- Aldehyde Dehydrogenase and Aldo-Keto Reductase Enzymes: Basic Concepts and Emerging Roles in Diabetic Retinopathy.Antioxidants (Basel, Switzerland) · 2023Review
- Adaptive Optics Imaging of Inherited Retinal Disease.Cold Spring Harbor perspectives in medicine · 2023Review
- Multimodal imaging reveals retinoschisis masquerading as retinal detachment in patients with choroideremia.American journal of ophthalmology case reports · 2022Article
- Reduced vessel density in deep capillary plexus correlates with retinal layer thickness in choroideremia.The British journal of ophthalmology · 2021Observational
- Inclusion of PF68 Surfactant Improves Stability of rAAV Titer when Passed through a Surgical Device Used in Retinal Gene Therapy.Molecular therapy. Methods & clinical development · 2020Article
- Adeno-Associated Viral Vectors in Neuroscience Research.Molecular therapy. Methods & clinical development · 2020Review
- DualHuman gene therapy · 2019Article
- Molecular Therapies for Choroideremia.Genes · 2019Review
- Adeno-Associated Viral Gene Therapy for Inherited Retinal Disease.Pharmaceutical research · 2019Review
- Choroideremia: Update On Clinical Features And Emerging Treatments.Clinical ophthalmology (Auckland, N.Z.) · 2019Review
- The Biological Activity of AAV Vectors for Choroideremia Gene Therapy Can Be Measured byMolecular therapy. Methods & clinical development · 2018Article
- Impact of Vital Dyes on Cell Viability and Transduction Efficiency of AAV Vectors Used in Retinal Gene Therapy Surgery: An In Vitro and In Vivo Analysis.Translational vision science & technology · 2017Article
- Inclusion of the Woodchuck Hepatitis Virus Posttranscriptional Regulatory Element Enhances AAV2-Driven Transduction of Mouse and Human Retina.Molecular therapy. Nucleic acids · 2017Article
- Review
- Leber's congenital amaurosis and the role of gene therapy in congenital retinal disorders.International journal of ophthalmology · 2017Review
- Retinal Gene Therapy: Surgical Vector Delivery in the Translation to Clinical Trials.Frontiers in neuroscience · 2017Review
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 2 institutions in 1 country.
Funding
Abstract
Choroideremia is an outer retinal degeneration with a characteristic clinical appearance that was first described in the nineteenth century. The disorder begins with reduction of night vision and gradually progresses to blindness by middle age. The appearance of the fundus in sufferers is recognizable by the characteristic pale color caused by the loss of the outer retina, retinal-pigmented epithelium, and choroidal vessels, leading to exposure of the underlying sclera. Choroideremia shows X-linked recessive inheritance and the choroideremia gene (CHM) was one of the first to be identified by positional cloning in 1990. Subsequent identification and characterization of the CHM gene, which encodes Rab escort protein 1 (REP1), has led to better comprehension of the disease and enabled advances in genetic diagnosis. Despite several decades of work to understand the exact pathogenesis, no established treatments currently exist to stop or even slow the progression of retinal degeneration in choroideremia. Encouragingly, several specific molecular and clinical features make choroideremia an ideal candidate for treatment with gene therapy. This work describes the considerations and challenges in the development of a new clinical trial using adeno-associated virus (AAV) encoding the CHM gene.
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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.