SynthesisDer Ophthalmologe : Zeitschrift der Deutschen Ophthalmologischen Gesellschaft2015
[Gene therapy as a treatment concept for inherited retinal diseases].
Synthesis in Der Ophthalmologe : Zeitschrift der Deutschen Ophthalmologischen Gesellschaft, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.
What it found
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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
3 citing papers in PubMed.
- Influence of Systematic Gaze Patterns in Navigation and Search Tasks with Simulated Retinitis Pigmentosa.Brain sciences · 2021Article
- Statement of the DOG, the RG, and the BVA on the therapeutic use of voretigene neparvovec (Luxturna™) in ophthalmology. English version : January 2019.Der Ophthalmologe : Zeitschrift der Deutschen Ophthalmologischen Gesellschaft · 2020Review
- [Statement of the German Society of Ophthalmology (DOG), the German Retina Society (RG) and the Professional Association of German Ophthalmologists (BVA) on the therapeutic use of voretigene neparvovec-rzyl (Luxturna™) in ophthalmology : Situation January 2019].Der Ophthalmologe : Zeitschrift der Deutschen Ophthalmologischen Gesellschaft · 2019Review
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
Abstract
backgroundGene therapy for inherited retinal diseases (IRDs) is currently being validated in several clinical trials and is becoming a promising therapeutic option for these previously incurable diseases.
objectivesThe aim of this review is to give an overview of the concept, the application and the challenges associated with gene therapy. In particular, the pertinence of gene therapy for IRDs will be highlighted along with ongoing clinical trials in the field. MATERIAL AND
methodsA systematic review of relevant entries on gene therapy and on gene therapy for IRDs, in particular in PubMed and ClinicalTrials.gov.
resultsGene therapy is emerging not only as a therapy for monogenetic retinal diseases but also for complex genetic diseases, such as neovascular age-related macular degeneration. The discovery of adeno-associated viral vectors (AAVs) has marked a great improvement for IRD gene therapy. All clinical studies since 2006 demonstrated the safety and initial efficacy; however, not all expectations based on very successful preclinical studies were met.
conclusionIn future we can expect gene therapy to continue to become more clinically relevant. More than ever, it is now essential to generate precise characterizations of the natural disease progression of IRDs through observational or retrospective studies in order to guarantee a most effective study design.
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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.