ReviewDer Ophthalmologe : Zeitschrift der Deutschen Ophthalmologischen Gesellschaft2021
[Experimental therapeutic approaches for the treatment of retinal dystrophy in neuronal ceroid lipofuscinosis].
Review in Der Ophthalmologe : Zeitschrift der Deutschen Ophthalmologischen Gesellschaft, 2021. 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, 3 citations in OpenAlex.
- [Therapeutic accessibility of the retina in systemic immunomodulatory approaches in mouse models for neuronal ceroid lipofuscinosis : Comments on Bartsch et al., Der Ophthalmologe 02/2021].Der Ophthalmologe : Zeitschrift der Deutschen Ophthalmologischen Gesellschaft · 2021Article
- [Preclinical developments of treatment options for retinal dystrophy in neuronal ceroid lipofuscinosis].Der Ophthalmologe : Zeitschrift der Deutschen Ophthalmologischen Gesellschaft · 2021Article
- Intracranial delivery of AAV9 gene therapy partially prevents retinal degeneration and visual deficits in CLN6-Batten disease mice.Molecular therapy. Methods & clinical development · 2021Article
Corrections and comments
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Authors and funding
6 authors at 3 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundNeuronal ceroid lipofuscinosis (NCL) is a group of rare and fatal neurodegenerative lysosomal storage diseases. Progressive retinal degeneration and loss of vision are among the characteristic symptoms of affected patients. A brain-directed enzyme replacement therapy has been shown to significantly attenuate the neurological symptoms in CLN2 patients and is currently the only approved therapy for NCL; however, there is presently no treatment option for retinal dystrophy in NCL.
objectiveThis short review aims to give an overview of preclinical studies that have developed and evaluated therapeutic strategies for the treatment of retinal dystrophy in animal models of different NCL forms. MATERIAL AND
methodsThe key findings of preclinical studies that have achieved positive therapeutic effects on retinal structure and/or function using different treatment strategies are summarized and discussed. RESULTS AND
conclusionThe published data on preclinical studies demonstrate the efficacy of different therapeutic strategies to attenuate retinal degeneration and vision loss in animal models for different NCL forms. It remains to be seen whether these promising results can be confirmed in future clinical studies.
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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.