ArticleExpert opinion on orphan drugs2018
Gene therapy for the treatment of X-linked retinitis pigmentosa.
Article in Expert opinion on orphan drugs, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 37 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
37 citing papers in PubMed, 54 citations in OpenAlex.
- Microperimetry Sensitivity Changes at the Functional Transition Zone and at the EZ Margins in X-Linked Retinitis Pigmentosa Treated With Gene Therapy.Translational vision science & technology · 2026Trial
- Three-Year Safety Results of SAR422459 (EIAV-ABCA4) Gene Therapy in Patients With ABCA4-Associated Stargardt Disease: An Open-Label Dose-Escalation Phase I/IIa Clinical Trial, Cohorts 1-5.American journal of ophthalmology · 2022Trial
- The quality-of-life impacts and economic burden of X-linked retinitis pigmentosa caused by variants in RPGR.Eye (London, England) · 2026Review
- Comparative Analysis of Retinal Structure and Function in Female Carriers of Choroideremia and X-Linked Retinitis Pigmentosa.Translational vision science & technology · 2026Article
- Valuing Maintenance of Visual Function in Retinitis Pigmentosa: A Discrete Choice Experiment with Patients and the General Public.PharmacoEconomics - open · 2026Article
- Content Validity of Two Generic Preference-Based Measures in Retinitis Pigmentosa, Including X-Linked Retinitis Pigmentosa.PharmacoEconomics - open · 2026Article
- Age-Stratified Genetic Spectrum of Retinitis Pigmentosa in Korean Patients: Predominance ofAnnals of laboratory medicine · 2026Article
- Functional and Structural Analysis of the Central Retina According to the Fundus Autofluorescence Pattern in Patients with Retinitis Pigmentosa.Diagnostics (Basel, Switzerland) · 2026Article
- Health state utilities associated with X-linked retinitis pigmentosa (XLRP).The European journal of health economics : HEPAC : health economics in prevention and care · 2025Article
- The Exponential Constriction Model of the Ellipsoid Zone in Taiwanese Individuals With RPGR-Related X-Linked Retinitis Pigmentosa.Investigative ophthalmology & visual science · 2025Article
- Genomic medicine and personalized treatment: a narrative review.Annals of medicine and surgery (2012) · 2025Review
- Pre-Clinical and Clinical Advances in Gene Therapy of X-Linked Retinitis Pigmentosa: Hope on the Horizon.Journal of clinical medicine · 2025Review
- Advancing vision: gene therapy innovations for X-linked retinitis pigmentosa (XLRP).EXCLI journal · 2025Article
- Early-Onset Cone Photoreceptor Degeneration Is Associated With High Myopia inJournal of ophthalmology · 2025Article
- Infantile Nystagmus Syndrome-Associated Inherited Retinal Diseases: Perspectives from Gene Therapy Clinical Trials.Life (Basel, Switzerland) · 2024Review
- Current developments of gene therapy in human diseases.MedComm · 2024Review
- Impacts of X-linked Retinitis Pigmentosa and Patient Pathways in European Countries: Results from the Cross-sectional EXPLORE XLRP-1 Physician Survey.Advances in therapy · 2024Article
- Deep Learning-Facilitated Study of the Rate of Change in Photoreceptor Outer Segment Metrics in RPGR-Related X-Linked Retinitis Pigmentosa.Investigative ophthalmology & visual science · 2023Article
- Translatability barriers between preclinical and clinical trials of AAV gene therapy in inherited retinal diseases.Vision research · 2023Review
- Clinical characteristics of high myopia in female carriers of pathogenicOphthalmic genetics · 2023Review
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 at 3 institutions in 3 countries.
Funding
Abstract
introductionX-linked retinitis pigmentosa caused by mutations in the AREAS COVERED: This manuscript reviews the natural history of X-linked retinitis pigmentosa and the research performed from the discovery of the causative gene, EXPERT OPINION: X-linked retinitis pigmentosa is an amenable disease to be treated by gene therapy. Codon optimisation has overcome the challenge of designing an
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.