ReviewCureus2024
Advances and Challenges in Gene Therapy for Inherited Retinal Dystrophies: A Comprehensive Review.
Review in Cureus, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 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
8 citing papers in PubMed.
- Disease and Participant-Related Correlates of Genetic Testing Completion for Hereditary Eye Disorders in a Cohort of over 1400 Patients.Ophthalmology science · 2026Article
- Exosome-based therapeutics: emerging extracellular vesicle platforms for targeted delivery in inherited retinal dystrophies and beyond.Annals of medicine and surgery (2012) · 2026Article
- Exploring the potential for gene therapy in Cav1.4-related retinal channelopathies.Channels (Austin, Tex.) · 2025Review
- Progress of iPSC-derived retinal organoids in the study of inherited retinal diseases.Orphanet journal of rare diseases · 2025Review
- Nanovesicular Drug Delivery Systems for Rare Ocular Diseases: Advances, Challenges, and Future Directions.AAPS PharmSciTech · 2025Review
- State of the Art on Inherited Retinal Dystrophies: Management and Molecular Genetics.Journal of clinical medicine · 2025Review
- Keep an Eye on Next Generation Sequencing (NGS) Technology: Secondary Findings and Differential Diagnosis in Inherited Retinal Dystrophies (IRDs).Biomedicines · 2025Review
- Rescue of the disease-associated phenotype in CRISPR-corrected hiPSCs as a therapeutic approach for inherited retinal dystrophies.Molecular therapy. Nucleic acids · 2025Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Inherited retinal dystrophies (IRDs) are a diverse group of genetic disorders leading to progressive vision loss due to the degeneration of retinal photoreceptors. Gene therapy has emerged as a promising approach to address the underlying genetic causes of IRDs, offering the potential for restoring vision and halting disease progression. This review provides a comprehensive overview of gene therapy innovations for IRDs, focusing on the mechanisms, recent advancements, and ongoing challenges. We discuss the fundamental principles of gene therapy, including the use of viral and non-viral vectors, and highlight key developments such as the approval of Luxturna for RPE65-mediated retinal dystrophy and the application of gene editing technologies like CRISPR/Cas9. Despite these advancements, significant challenges remain, including vector delivery, long-term safety, and variable patient responses. This review also explores the future directions of gene therapy, emphasizing the need for further research to address these challenges and enhance therapeutic efficacy. By examining the current state of gene therapy for IRDs, this review aims to provide valuable insights into the potential for these treatments to transform the management of retinal diseases and improve the quality of life for affected individuals.
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.