ReviewInvestigative ophthalmology & visual science2011
AAV and compacted DNA nanoparticles for the treatment of retinal disorders: challenges and future prospects.
Review in Investigative ophthalmology & visual science, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 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
21 citing papers in PubMed.
- Investigating PRPH2-associated retinal degeneration patient-reported outcomes in vision-related quality of life.BMC ophthalmology · 2026Article
- Nanoparticles-mediated CRISPR-Cas9 gene therapy in inherited retinal diseases: applications, challenges, and emerging opportunities.Journal of nanobiotechnology · 2022Review
- Electrospun-Fibrous-Architecture-Mediated Non-Viral Gene Therapy Drug Delivery in Regenerative Medicine.Polymers · 2022Review
- The Next Generation of Molecular and Cellular Therapeutics for Inherited Retinal Disease.International journal of molecular sciences · 2021Review
- Distribution of polymeric nanoparticles in the eye: implications in ocular disease therapy.Journal of nanobiotechnology · 2021Review
- Treatment-Emergent Adverse Events in Gene Therapy Trials for Inherited Retinal Diseases: A Narrative Review.Ophthalmology and therapy · 2020Review
- "Para-retinal" Vector Administration into the Deep Vitreous Enhances Retinal Transgene Expression.Molecular therapy. Methods & clinical development · 2020Article
- Voretigene Neparvovec in Retinal Diseases: A Review of the Current Clinical Evidence.Clinical ophthalmology (Auckland, N.Z.) · 2020Review
- Comparison of structural progression between ciliopathy and non-ciliopathy associated with autosomal recessive retinitis pigmentosa.Orphanet journal of rare diseases · 2019Article
- Lipid nanoparticles for delivery of messenger RNA to the back of the eye.Journal of controlled release : official journal of the Controlled Release Society · 2019Article
- Gene Editing Preserves Visual Functions in a Mouse Model of Retinal Degeneration.Frontiers in neuroscience · 2019Article
- Therapeutic Benefits from Nanoparticles: The Potential Significance of Nanoscience in Retinal Degenerative Diseases.Journal of molecular biology & therapeutics · 2019Article
- Nanoparticles as Delivery Vehicles for the Treatment of Retinal Degenerative Diseases.Advances in experimental medicine and biology · 2018Review
- Review
- Nanoparticle-mediated miR200-b delivery for the treatment of diabetic retinopathy.Journal of controlled release : official journal of the Controlled Release Society · 2016Article
- Progress in corneal wound healing.Progress in retinal and eye research · 2015Review
- Nanoparticle-based technologies for retinal gene therapy.European journal of pharmaceutics and biopharmaceutics : official journal of Arbeitsgemeinschaft fur Pharmazeutische Verfahrenstechnik e.V · 2015Review
- Lipid Nanoparticles for Ocular Gene Delivery.Journal of functional biomaterials · 2015Review
- Gene therapy for PRPH2-associated ocular disease: challenges and prospects.Cold Spring Harbor perspectives in medicine · 2014Review
- Gene therapy in animal models of autosomal dominant retinitis pigmentosa.Molecular vision · 2012Review
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.
Funding
Abstract
Gene therapy based on delivery of viral and nonviral vectors has shown great promise for the treatment of human ocular diseases; however, limitations have consistently prevented its widespread clinical application. Viral vectors have generally been better in terms of efficiency but have safety concerns. Nonviral vectors, on the other hand, offer safety but have often been disappointing in terms of efficiency of nuclear delivery and gene expression. Extensive animal studies have reported significant progress using both systems, but thus far only a few studies have shown promise in human clinical trials. This article reviews both viral and nonviral work with focus on two candidates for clinical ocular application--AAV and nanoparticles. Of particular interest are various requirements for successful clinical application of these technologies including vector trafficking, delivery, specific gene expression, and treatment safety, and tolerance.
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.